Detailed Phase 3 CANOPY-HCH-3 data in children living with hypochondroplasia also featured in a late-breaking oral presentation at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting
VOXZOGO demonstrated statistically significant improvements across multiple measures of growth, including annualized growth velocity, standing height, height Z-score and arm span
BioMarin recently submitted a supplemental New Drug Application (sNDA) to the FDA to support expanding treatment with VOXZOGO to include children with hypochondroplasia
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO® (vosoritide) in children with hypochondroplasia were published in New England Journal of Medicine (NEJM) Evidence and presented at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting. The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualized growth velocity (AGV), standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia.
The CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p<0.0001). Children treated with VOXZOGO also showed significant improvements in standing height (LS mean difference of 2.35 cm; p<0.0001), height Z-score (LS mean difference of 0.39 standard deviation score; p<0.0001), and arm span (LS mean difference of 1.03 cm; p=0.0082) compared with placebo. Children who received VOXZOGO also demonstrated numerical improvements in quality of life, and follow-up will continue to assess the impact of treatment over a longer term. The overall safety profile was consistent with previous studies of VOXZOGO, with most adverse events reported as mild and no treatment-related serious adverse events identified.
"These results presented in detail for the first time provide a comprehensive picture of the impact of VOXZOGO across multiple measures of growth in children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Based on this compelling body of evidence, we have submitted these data to the FDA with the goal of securing approval for the first medicine for children with hypochondroplasia."
"Hypochondroplasia can affect a child's growth, physical function and everyday life, with families often navigating unique challenges as they support their children's development," said Andrew Dauber, M.D., lead study investigator and Chief of Endocrinology at Children's National in Washington, D.C. "The changes we observed in annualized growth velocity and arm span provide encouraging evidence of how children with hypochondroplasia responded to treatment throughout the study. These findings deepen our understanding of the condition while reinforcing VOXZOGO's potential as the first targeted medicine developed specifically for children with hypochondroplasia."
BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia and are on track with the submissions to the European Medicines Agency (EMA) and other regional health authorities. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch.
Below are key BioMarin presentations across both achondroplasia and hypochondroplasia at ESPE, with all times listed in Central European Summer Time:
Vosoritide Increases Growth Velocity in Children With Hypochondroplasia: Phase 3 Trial Results
Oral Presentation #LBA 1067
Wednesday, Sept. 9, 10:48 – 10:56 a.m.
Vosoritide Safety and Effectiveness in Young Children With Achondroplasia Aged ≤3 Years and With up to 36 Months of Follow-Up from the Japanese Post-Marketing Safety Surveillance Study (111-604)
Oral Presentation #FC3.4
Tuesday, Sept. 8, 3:30 – 3:40 p.m.
About Hypochondroplasia
Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO.
There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia.
For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.
About VOXZOGO
In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.
VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). An sNDA with long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth such as body proportionality and arm span is under review with an FDA Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.
The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency.
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO?
VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO?
The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken?
VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO?
Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see additional safety information in the full Prescribing Information and Patient Information.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting, including the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia and achondroplasia; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including the expectation that, if approved by the U.S. Food and Drug Administration (FDA), VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia with a potential 2027 launch; BioMarin's expectations regarding its supplemental New Drug Application (sNDA) for VOXZOGO for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; and BioMarin's estimate regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
Key Takeaways BioMarin will receive royalties on Yuviwel sales, turning a patent dispute into a royalty stream.BMRN gets 20% U.S. royalties and 18% in the EU, Brazil and South Korea through May 2030.Yuviwel reached 8 million euros in second-quarter revenue and enrolled more than 220 patients by July 31. For BioMarin Pharmaceutical (BMRN - Free Report) , the FDA approval of Ascendis Pharma’s (ASND - Free Report) Yuviwel in February had created a problem — a new entrant targeting the same achondroplasia market as Voxzogo, its key revenue driver. But a recent settlement between the two companies has changed the economics of that competition in a meaningful way.
Rather than continue fighting a competitor that had already entered the market, BioMarin has found a way to make the competitor pay it. Under the settlement, Ascendis will pay BioMarin royalties on Yuviwel sales in the United States, European Union, Brazil and South Korea, effectively giving BMRN an economic interest in a drug that could otherwise take share from Voxzogo.
BMRN Turns a Patent Dispute Into a Royalty StreamWhen Voxzogo secured FDA approval in 2021, it became the first FDA-approved therapy for achondroplasia, the most common form of dwarfism. The drug is a C-type natriuretic peptide (CNP) analog administered once daily as a subcutaneous injection. CNP is a naturally occurring signaling molecule involved in bone growth.
When Ascendis launched Yuviwel in April, its entry was a significant challenge for BioMarin, as Voxzogo had previously been the only approved treatment for achondroplasia. Like Voxzogo, Yuviwel is also a CNP therapy but is administered once weekly. The ASND drug is designed to provide continuous exposure to active CNP, potentially offering a more convenient dosing profile than the BMRN drug.
BioMarin had alleged that Yuviwel infringed its intellectual property covering CNP technology, triggering patent disputes across the United States, Brazil, Denmark, Germany and South Korea, including before the U.S. International Trade Commission. But instead of continuing to fight, both companies have settled their disputes with a licensing deal.
Per the settlement terms, BioMarin will grant Ascendis a non-exclusive, worldwide license to certain patents covering Yuviwel and related products, allowing Ascendis to research, develop, manufacture and commercialize the drug across current and potential indications.
In return, Ascendis will pay BioMarin royalties equal to 20% of annual net sales in the United States and 18% in the European Union, Brazil and South Korea, from the first commercial sale in each territory through May 20, 2030. The U.S. royalty is also applied retroactively to Yuviwel's first commercial sale. All pending lawsuits between the two companies will also be dismissed.
Competitive Threat for BMRN Still RemainsThe settlement changes the competitive equation for BioMarin. Previously, every dollar of Yuviwel sales represented a potential dollar of revenue at risk for Voxzogo. Under the new agreement, Yuviwel sales can also generate revenues for BioMarin through royalties. This means that while greater adoption of Yuviwel could still put pressure on Voxzogo, BMRN will now participate in the growth of its competitor.
The opportunity is becoming more meaningful as Yuviwel gains traction. Ascendis generated €8 million in Yuviwel revenues in the second quarter of 2026, its first quarter of launch, and had enrolled more than 220 patients through July 31. More than 100 healthcare providers had prescribed the drug, with more than 65% of enrollments approved for reimbursement. Ascendis is also preparing for further expansion, with its EU filings under review and additional studies underway.
Still, the settlement does not eliminate the competitive risk for BioMarin. Royalties are only a partial offset to any erosion in Voxzogo sales, and the economics will ultimately depend on how quickly Yuviwel penetrates the market and how much of that growth comes at Voxzogo’s expense.
BioMarin’s Voxzogo could have another potential competitor by this time next year. Recently, BridgeBio Pharma (BBIO - Free Report) has submitted a regulatory filing with the FDA for infigratinib, an FGFR3 inhibitor that could become the first approved oral therapy for achondroplasia. The company expects a U.S. launch in mid-2027, subject to approval.
The arrival of another potentially differentiated therapy underscores that BioMarin’s competitive challenge is unlikely to end with Yuviwel. The company may have found a clever way to monetize one threat, but it will still need to defend Voxzogo as the achondroplasia market becomes increasingly competitive.
BMRN’s Price Performance, Valuation & EstimatesShares of BioMarin have risen 13% year to date, significantly outperforming the industry’s 11% growth.
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From a valuation standpoint, the company is currently trading at a premium to the industry. Based on the price-to-sales (P/S) ratio, the stock trades at 3.06 times forward 12-month sales per share, higher than the industry average of 1.94 times.
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Movements in EPS estimates for 2026 and 2027 have been mixed over the past 30 days.
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BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Wells Fargo 21st Annual Healthcare Conference, September 9 at 3:00 pm EST Cantor Global Healthcare Conference 2026, September 10 at 10:20 am EST Citi's 2026 Biopharma Back to School Conference, September 10 at 1:00 pm EST Morgan Stanley 24th Annual Global Healthcare Conference, September 14 at 10:00 am EST SAN RAFAEL, Calif., Sept. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that management will present at upcoming investor conferences in September.
Acquisition adds ALE1, a potential first oral therapy for hypophosphatasia to clinical pipeline
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) said today that it completed the previously announced agreement to acquire Alesta Therapeutics. The acquisition will strengthen BioMarin's skeletal conditions portfolio, adding ALE1, an oral small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. ALE1 is currently being evaluated in an ongoing Phase 1/2a clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.
On August 18, BioMarin announced it had entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta's lead clinical-stage asset, ALE1. Prior to the close of the transaction, Alesta spun out all non-ALE1 assets to a new entity. Alesta Therapeutics is now a wholly owned subsidiary of BioMarin.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.
To learn more, please visit www.biomarin.com.
About Alesta Therapeutics
Alesta Therapeutics is a clinical-stage biotechnology company committed to developing novel oral small-molecule therapies for underserved diseases. The company's lead asset, ALE1, is being developed for hypophosphatasia (HPP), a rare genetic disorder with significant unmet need.
For more information, visit www.alestatherapeutics.com.
Forward-Looking Statements
This press release contains forward-looking statements about, among other things, the acquisition of ALE1, the lead clinical-stage asset, of Alesta Therapeutics (Alesta) by BioMarin Pharmaceutical Inc. (BioMarin) and the business prospects of BioMarin, including, without limitation, statements about: the prospective benefits of the acquisition, including expectations that it will strengthen BioMarin's skeletal conditions portfolio; expectations regarding ALE1 and its ongoing development, including its potential to be a first-in-class oral therapy for the treatment of hypophosphatasia (HPP); and other statements that are not historical facts.
These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the effects of the acquisition (or the announcement thereof) on BioMarin's stock price and/or BioMarin's operating results; unknown or inestimable liabilities; BioMarin's ability to successfully develop, launch and commercialize products and product candidates such as ALE1, if approved; BioMarin's ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period and that integration will not be successful or that such integration may be more difficult, time-consuming or costly than expected; the time-consuming and uncertain regulatory approval process for pharmaceutical product development; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. or its affiliates.
Agreement Resolves all Pending Patent-Related Proceedings Between BioMarin and Ascendis
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has entered into binding terms with Ascendis Pharma A/S, resolving the patent and ancillary disputes pending globally, including before the U.S. International Trade Commission (ITC) concerning Ascendis's Yuviwel. As part of the agreement, Ascendis will pay BioMarin a royalty equal to 20% of net sales of Yuviwel in the U.S., retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil and South Korea until May 2030.
"This outcome incentivizes companies like BioMarin to keep investing in the kind of long-term innovation that is critical to bringing breakthrough treatments to the people who need them," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We have spent decades focused on understanding the underlying biology of rare genetic conditions, building the deep scientific expertise that led to our development of six first-in-disease medicines for patients. We look forward to continuing to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on our ongoing momentum for children with achondroplasia."
The scope of the settlement includes a license for BioMarin's patents that relate to Yuviwel for all current and potential indications, including achondroplasia and hypochondroplasia. It also covers the use of Yuviwel in combination with other medicines. Under the terms of the agreement, BioMarin will dismiss the pending Section 337 investigation before the ITC and the parties will resolve all claims relating to the asserted intellectual property, including litigation pending in Brazil, Denmark, Germany, South Korea and the Northern District of California.
Reaching this agreement recognizes the value of BioMarin's pioneering innovations in C-type natriuretic peptide (CNP) technology, including the development of VOXZOGO® (vosoritide), while providing a framework that enables continued access to medicine for children with achondroplasia around the world.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the settlement and license agreement with Ascendis Pharma A/S, including expected benefits of such agreement and anticipated royalty payments, and future commercialization of licensed products and BioMarin's expectations to continue to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on its momentum for children with achondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's ability to enforce the agreement; actual sales of licensed products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
On Sunday, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) reached a binding settlement with Ascendis Pharma A/S (NASDAQ:ASND)to resolve all global patent disputes regarding the medication Yuviwel.
Ascendis Pharma’s Yuviwel (navepegritide, developed as TransCon CNP) is a once-weekly prescription injection approved by the FDA to increase linear height growth in children aged 2 years and older with achondroplasia who have open growth plates.
In March, the FDA approved Yuviwel, the first and only once-weekly treatment for children with achondroplasia.
Ends Global Legal DisputesThe sweeping agreement ends ongoing legal conflicts and intellectual property challenges between the two pharmaceutical companies, including a U.S. International Trade Commission (ITC) investigation.
Under the newly established financial terms, Ascendis will pay BioMarin a 20% royalty on all net sales of Yuviwel within the U.S.
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The companies agreed to apply this royalty rate retroactively, covering all transactions dating back to the drug’s initial commercial sale.
Additionally, BioMarin will collect an 18% royalty on net sales generated across the European Union, Brazil, and South Korea. These international royalty payments will remain active and continue through May 2030.
Ascendis Receives License For BioMarin PatentsThe settlement grants Ascendis a full license to utilize BioMarin’s patents associated with Yuviwel. This comprehensive license covers all current and potential medical indications, specifically including hypochondroplasia and achondroplasia.
Furthermore, the agreement permits the use of Yuviwel in combination treatments involving other medicines.
Both parties will also dismiss all related intellectual property claims and lawsuits currently active in the Northern District of California, Germany, Denmark, South Korea, and Brazil.
Ascendis Targets 500 Million Euros In 2026 Operating Cash FlowAs previously communicated, Ascendis expects to generate more than 500 million euros in operating cash flow in 2026 and believes that it can achieve 5 billion euros in revenue in 2030.
Yuviwel revenue for the second quarter of 2026 totaled 8 million euros.
More than 220 unique Yuviwel patient enrollments by more than 100 prescribing healthcare providers, with more than 65% of enrollments approved for reimbursement in the U.S. through July 31, 2026.
Yuviwel European Review Remains UnderwayMarketing Authorization Application remains under review by the European Medicines Agency, with a decision anticipated in the fourth quarter of 2026.
Ascendis Pharma expects to initiate enrollment in a Phase 3 trial in the second half of the year to investigate TransCon CNP monotherapy for hypochondroplasia.
Price Action: Ascendis Pharma shares were down 0.21% at $247.40 and BioMarin Pharmaceutical shares were up 3.33% at $66.81 during premarket trading on Monday, according to Benzinga Pro data.
ADAR1 Capital Management LLC acquired a new stake in BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) during the 2nd quarter, according to the company in its most recent disclosure with the Securities and Exchange Commission. The institutional investor acquired 49,000 shares of the biotechnology company’s stock, valued at approximately $2,804,000.
Other hedge funds and other institutional investors have also bought and sold shares of the company. NewEdge Advisors LLC boosted its position in BioMarin Pharmaceutical by 11.5% in the 1st quarter. NewEdge Advisors LLC now owns 3,769 shares of the biotechnology company’s stock valued at $266,000 after buying an additional 388 shares during the period. Empowered Funds LLC increased its position in shares of BioMarin Pharmaceutical by 207.4% during the first quarter. Empowered Funds LLC now owns 12,023 shares of the biotechnology company’s stock worth $850,000 after acquiring an additional 8,112 shares during the period. Focus Partners Wealth raised its stake in shares of BioMarin Pharmaceutical by 70.2% during the first quarter. Focus Partners Wealth now owns 11,817 shares of the biotechnology company’s stock valued at $835,000 after acquiring an additional 4,876 shares in the last quarter. Sivia Capital Partners LLC increased its holdings in BioMarin Pharmaceutical by 33.0% during the 2nd quarter. Sivia Capital Partners LLC now owns 4,439 shares of the biotechnology company’s stock worth $244,000 after purchasing an additional 1,102 shares during the period. Finally, Invesco Ltd. raised its position in BioMarin Pharmaceutical by 23.5% during the 2nd quarter. Invesco Ltd. now owns 618,835 shares of the biotechnology company’s stock valued at $34,017,000 after purchasing an additional 117,617 shares in the last quarter. Hedge funds and other institutional investors own 98.71% of the company’s stock.
Analyst Ratings Changes BMRN has been the topic of a number of recent research reports. Sanford C. Bernstein dropped their price target on BioMarin Pharmaceutical from $94.00 to $82.00 and set an “outperform” rating on the stock in a research report on Tuesday, May 5th. Canaccord Genuity Group lifted their price objective on BioMarin Pharmaceutical from $111.00 to $114.00 and gave the company a “buy” rating in a report on Monday, August 10th. Royal Bank Of Canada lowered their price objective on shares of BioMarin Pharmaceutical from $66.00 to $62.00 and set a “sector perform” rating for the company in a research note on Tuesday, July 7th. Bank of America dropped their target price on shares of BioMarin Pharmaceutical from $85.00 to $80.00 and set a “buy” rating on the stock in a report on Tuesday, May 19th. Finally, The Goldman Sachs Group assumed coverage on shares of BioMarin Pharmaceutical in a research report on Monday, May 11th. They set a “neutral” rating and a $69.00 target price on the stock. Two analysts have rated the stock with a Strong Buy rating, fifteen have given a Buy rating, six have given a Hold rating and one has issued a Sell rating to the stock. According to MarketBeat, the company has an average rating of “Moderate Buy” and a consensus price target of $88.83.
Check Out Our Latest Analysis on BMRN BioMarin Pharmaceutical Stock Performance Shares of BMRN stock opened at $66.12 on Wednesday. BioMarin Pharmaceutical Inc. has a 12-month low of $49.26 and a 12-month high of $70.98. The business’s fifty day simple moving average is $61.07 and its 200-day simple moving average is $57.99. The company has a current ratio of 2.39, a quick ratio of 1.32 and a debt-to-equity ratio of 0.56. The company has a market cap of $12.80 billion, a PE ratio of 178.70, a PEG ratio of 0.47 and a beta of 0.24.
BioMarin Pharmaceutical (NASDAQ:BMRN – Get Free Report) last posted its quarterly earnings data on Thursday, August 6th. The biotechnology company reported $1.20 earnings per share for the quarter, topping analysts’ consensus estimates of $0.95 by $0.25. The firm had revenue of $989.71 million during the quarter, compared to analysts’ expectations of $932.03 million. BioMarin Pharmaceutical had a return on equity of 5.32% and a net margin of 2.14%.The business’s quarterly revenue was up 20.0% on a year-over-year basis. During the same quarter last year, the firm earned $1.44 EPS. BioMarin Pharmaceutical has set its FY 2026 guidance at 4.900-5.100 EPS. Analysts expect that BioMarin Pharmaceutical Inc. will post 4.1 earnings per share for the current fiscal year.
BioMarin Pharmaceutical Company Profile (Free Report)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
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Bank of New York Mellon Corp bought a new stake in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) in the second quarter, according to the company in its most recent Form 13F filing with the Securities and Exchange Commission (SEC). The institutional investor bought 1,155,305 shares of the biotechnology company’s stock, valued at approximately $66,107,000. Bank of New York Mellon Corp owned about 0.60% of BioMarin Pharmaceutical at the end of the most recent reporting period.
Other hedge funds and other institutional investors have also made changes to their positions in the company. CIBC Private Wealth Group LLC increased its position in shares of BioMarin Pharmaceutical by 38.2% during the fourth quarter. CIBC Private Wealth Group LLC now owns 648 shares of the biotechnology company’s stock worth $39,000 after purchasing an additional 179 shares in the last quarter. Northwestern Mutual Investment Management Company LLC lifted its holdings in BioMarin Pharmaceutical by 0.4% in the 4th quarter. Northwestern Mutual Investment Management Company LLC now owns 42,982 shares of the biotechnology company’s stock valued at $2,554,000 after purchasing an additional 183 shares in the last quarter. Caerus Investment Advisors LLC boosted its stake in BioMarin Pharmaceutical by 2.8% during the 4th quarter. Caerus Investment Advisors LLC now owns 10,236 shares of the biotechnology company’s stock worth $608,000 after purchasing an additional 278 shares during the last quarter. Chicago Partners Investment Group LLC boosted its stake in BioMarin Pharmaceutical by 2.4% during the 1st quarter. Chicago Partners Investment Group LLC now owns 12,183 shares of the biotechnology company’s stock worth $660,000 after purchasing an additional 290 shares during the last quarter. Finally, Parallel Advisors LLC grew its holdings in shares of BioMarin Pharmaceutical by 62.2% during the third quarter. Parallel Advisors LLC now owns 769 shares of the biotechnology company’s stock worth $42,000 after buying an additional 295 shares in the last quarter. Hedge funds and other institutional investors own 98.71% of the company’s stock.
Analysts Set New Price Targets A number of equities research analysts have recently commented on BMRN shares. Citigroup increased their target price on shares of BioMarin Pharmaceutical from $75.00 to $76.00 and gave the company a “buy” rating in a research report on Thursday, July 16th. Oppenheimer boosted their price target on shares of BioMarin Pharmaceutical from $95.00 to $97.00 and gave the stock an “outperform” rating in a research report on Friday, August 7th. Sanford C. Bernstein dropped their price objective on shares of BioMarin Pharmaceutical from $94.00 to $82.00 and set an “outperform” rating for the company in a research note on Tuesday, May 5th. Canaccord Genuity Group increased their price objective on shares of BioMarin Pharmaceutical from $111.00 to $114.00 and gave the company a “buy” rating in a report on Monday, August 10th. Finally, Wall Street Zen raised shares of BioMarin Pharmaceutical from a “hold” rating to a “buy” rating in a research note on Saturday, August 8th. Two equities research analysts have rated the stock with a Strong Buy rating, fifteen have assigned a Buy rating, six have issued a Hold rating and one has given a Sell rating to the company. Based on data from MarketBeat, the company has an average rating of “Moderate Buy” and an average target price of $88.79.
View Our Latest Stock Analysis on BMRN BioMarin Pharmaceutical Trading Down 0.8% Shares of BMRN opened at $65.61 on Thursday. BioMarin Pharmaceutical Inc. has a 12-month low of $49.26 and a 12-month high of $70.98. The firm has a market capitalization of $12.70 billion, a P/E ratio of 177.32, a P/E/G ratio of 0.47 and a beta of 0.24. The firm has a 50-day moving average price of $61.29 and a 200 day moving average price of $58.05. The company has a current ratio of 2.39, a quick ratio of 1.32 and a debt-to-equity ratio of 0.56.
BioMarin Pharmaceutical (NASDAQ:BMRN – Get Free Report) last issued its earnings results on Thursday, August 6th. The biotechnology company reported $1.20 EPS for the quarter, topping analysts’ consensus estimates of $0.95 by $0.25. The company had revenue of $989.71 million for the quarter, compared to the consensus estimate of $932.03 million. BioMarin Pharmaceutical had a net margin of 2.14% and a return on equity of 5.32%. BioMarin Pharmaceutical’s quarterly revenue was up 20.0% compared to the same quarter last year. During the same period in the previous year, the firm earned $1.44 earnings per share. BioMarin Pharmaceutical has set its FY 2026 guidance at 4.900-5.100 EPS. Sell-side analysts forecast that BioMarin Pharmaceutical Inc. will post 4.1 EPS for the current year.
(Free Report)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
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BioMarin Pharmaceutical is making more waves on the M&A front. The company announced Tuesday that it will acquire Alesta Therapeutics for $275 million upfront. The deal gives BioMarin access to Alesta's oral treatment for hypophosphatasia, or HPP – a rare genetic bone disease. The drug, ALE1, is currently in early stage clinical trials, but has the potential to be the first oral treatment for HPP. BioMarin will pay up to an additional $215 million if the drug hits certain milestones.
The move further bolsters BioMarin's unit focused on rare skeletal conditions, a portfolio anchored by its drug Voxzogo. It also marks the company's third major deal in the rare disease space over the past year, headlined by the $4.8 billion deal to buy Amicus Therapeutics announced last December.
"This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets – adding an asset that has the potential to reach our largest addressable patient population. We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin," BioMarin's president and CEO, Alexander Hardy, said in a statement released yesterday.
The release also noted that more than 9,000 people in the United States have been diagnosed with HPP, though it is believed that the true number of people impacted by the disease is likely higher.
Ilan Ganot, Alesta's CEO, said BioMarin's ability to scale the treatment factored into his board's decision to approve the deal. "BioMarin's global reach, scale, and proven expertise in rare disease drug development make it an ideal partner to advance ALE1 and realize its potential as a promising treatment for patients with HPP worldwide."
Key Takeaways BioMarin will acquire ALE1 for up to $490M, adding a potential oral therapy for rare HPP.The deal could broaden BioMarin's skeletal-disease portfolio beyond Voxzogo.ALE1 may offer a more convenient option than Strensiq, the established injectable HPP treatment. BioMarin Pharmaceutical (BMRN - Free Report) announced that it has entered into a definitive agreement to acquire clinical-stage biotech Alesta Therapeutics for up to $490 million. This includes an upfront payment of $275 million and the remaining $215 million in development and regulatory milestone payments.
Under the deal structure, BioMarin is effectively acquiring Alesta's lead clinical-stage asset, ALE1, rather than the company's entire existing portfolio. The drug is being developed in a phase I/IIa study as a potential treatment for hypophosphatasia (HPP), a rare genetic bone disease. If left untreated, HPP can lead to impaired bone and tooth mineralization, fractures, muscle weakness, fatigue and pain.
Immediately before the transaction closes, Alesta will spin out all non-ALE1 assets into a new entity, with Alesta employees transferring to the spinout. BioMarin will therefore gain ALE1 while avoiding the acquisition of the other assets and workforce associated with Alesta.
The boards of directors of both companies have approved the deal, which is expected to close in the third quarter of 2026, subject to customary closing conditions. BioMarin plans to fund the transaction with cash on hand. Although the company did not provide the financial impact of this transaction on its 2026 adjusted EPS, it expects the deal to have a modestly dilutive impact.
BioMarin to Broaden Rare Bone Disease PortfolioThe acquisition would expand BioMarin’s skeletal-disease portfolio, which is currently anchored by Voxzogo, the company’s largest product by net sales. The drug is on track to achieve blockbuster status in sales this year. Adding an HPP program would give the company exposure to another rare bone disorder and broaden its presence across the skeletal conditions market.
ALE1 is designed to target inorganic pyrophosphate (PPi), a metabolite central to HPP, with the potential to address both skeletal and broader disease manifestations through systemic correction of the underlying biology. The drug could also potentially offer a differentiated treatment approach in HPP, given its oral formulation. This could provide a more convenient alternative to the blockbuster therapy Strensiq, the established HPP treatment marketed by AstraZeneca (AZN - Free Report) through its Alexion division and administered via subcutaneous injection.
The commercial opportunity is significant, with Strensiq generating $1.05 billion in sales in the first half of 2026, up 41% year over year, driven by continued demand from HPP patients. The product’s performance highlights the established demand for HPP treatment that BioMarin could potentially address with ALE1.
BMRN Stock PerformanceShares of BioMarin were up 0.5% on Tuesday following the acquisition announcement. The deal was broadly consistent with management’s previously stated business-development strategy.
BioMarin completed the Amicus Therapeutics acquisition in April, which added two new marketed products to its portfolio — Galafold (for Fabry disease) and Pombiliti-Opfolda (a combination therapy for Pompe disease). During its second-quarter 2026 earnings call, management said the integration had strengthened and diversified its commercial portfolio, allowing the company to shift its focus toward expanding its clinical-stage pipeline. It added that BioMarin plans to pursue deals to expand its clinical-stage programs over the next 12 to 18 months.
Year to date, shares of BMRN have gained 13% compared with the industry’s 6% growth.
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BioMarin Bolsters Pipeline After Recent SetbackThe Alesta acquisition comes at a time when BioMarin seeks to replenish its clinical-stage pipeline following a setback with BMN 401, an investigational enzyme replacement therapy (ERT). In May, BioMarin announced mixed results from the phase III ENERGY 3 study, which evaluated this therapy for a rare genetic disorder called ENPP1 deficiency in children aged one to 12 years. The study did not meet one of its two main goals and failed to show positive trends across secondary endpoints.
BMN 401 was also being developed for two other rare disorders, but the company recently announced that it is discontinuing development of the therapy across all indications.
The decision represents a significant setback for the company, which acquired the therapy through its $270 million acquisition of Inozyme Pharma last year. BMN 401, formerly known as INZ-701, was the lead asset in Inozyme’s pipeline and was already in phase III development for ENPP1 deficiency when BioMarin acquired the company.
BMRN’s Zacks RankBioMarin currently carries a Zacks Rank #3 (Hold).
Our Key Picks Among Biotech StocksSome better-ranked stocks in the biotech sector are Anika Therapeutics (ANIK - Free Report) and Repligen Corporation (RGEN - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings per share (EPS) estimates for Anika Therapeutics have risen from 41 cents to $1.05 for 2026. Over the same period, EPS estimates have increased from 46 cents to 95 cents for 2027. ANIK shares have skyrocketed 124% year to date.
Anika Therapeutics missed on earnings in each of the trailing four quarters, delivering an average surprise of 950%.
Over the past 30 days, estimates for Repligen’s 2026 EPS have increased to $2.06 from $1.99. Over the same period, EPS estimates for 2027 have risen from $2.57 to $2.61. RGEN shares have gained 1% so far this year.
Repligen’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 16.80%.
BioMarin to pay $275 million upfront, plus additional payments upon achievement of development and regulatory milestones
Alesta to spin out all non-ALE1 assets to a new entity and Alesta employees to transfer to the spinout entity prior to close
ALE1 has significant potential to help BioMarin expand into larger rare disease markets with a medicine intended to address a high unmet need for patients and offers strong strategic fit for the company
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that BioMarin has entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta's lead clinical-stage asset, ALE1. Alesta plans to spin out all non-ALE1 assets prior to the close of the transaction, which has been approved by the boards of directors of both companies and is expected to be completed this quarter, subject to customary closing conditions.
ALE1 is an orally active, small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene. ALE1 has the potential to be the first oral therapy for HPP and is currently being evaluated in an ongoing Phase 1/2a clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP.
HPP is a serious condition that can affect bone and tooth mineralization, resulting in easy or frequent bone breaks, early tooth loss, and, in adults, clinically significant muscle weakness, fatigue and pain. If approved, ALE1 is expected to be the first oral therapy approach that targets the central disease metabolite, PPi (inorganic pyrophosphate), with the potential to impact both skeletal and broader manifestations of HPP through systemic correction of disease biology. The program will become part of BioMarin's Skeletal Conditions Business Unit following close.
"ALE1 is a strong strategic fit for BioMarin, bringing a potential oral alternative to the injectable therapies available today for people living with HPP around the world while meaningfully strengthening our early-stage clinical pipeline," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets – adding an asset that has the potential to reach our largest addressable patient population. We plan to continue to seek these kinds of opportunities as we focus on clinical-stage innovation to drive durable growth for BioMarin."
Under the terms of the agreement, BioMarin will acquire Alesta and Alesta shareholders will receive an upfront payment of $275 million plus up to $215 million in subsequent payments upon achievement of certain development and regulatory milestones. Additionally, immediately prior to the close of BioMarin's acquisition of Alesta, Alesta will spin out all non-ALE1 assets to a new entity and former Alesta employees will transfer to the spinout entity. As a result, no Alesta employees will become employees of BioMarin in connection with the transaction. BioMarin intends to fund the transaction with cash on hand. BioMarin expects to provide updated full-year 2026 guidance reflecting the acquisition of Alesta following the closing of the transaction. Excluding the upfront consideration, the transaction is expected to have a modestly dilutive impact on 2026 financial results.
"We chose to partner with BioMarin due to their deep commitment to people living with rare diseases," said Ilan Ganot, Chief Executive Officer of Alesta Therapeutics. "BioMarin's global reach, scale, and proven expertise in rare disease drug development make it an ideal partner to advance ALE1 and realize its potential as a promising treatment for patients with HPP worldwide. This acquisition is also a testament to the extraordinary work, scientific expertise, and drug development capabilities of the Alesta team."
Morgan Stanley & Co. LLC is acting as the exclusive financial advisor to BioMarin, and Jones Day is serving as legal counsel in connection with the acquisition. J.P. Morgan Securities LLC is acting as exclusive financial advisor to Alesta, and Goodwin Procter LLP and NautaDutilh N.V. are serving as legal counsel.
About ALE1
ALE1 is designed to inhibit a novel target that regulates levels of inorganic pyrophosphate (PPi), the metabolite at the center of HPP pathology. By lowering excess PPi, ALE1 aims to restore healthier bone and mineral metabolism across the full spectrum of HPP.
More than 9,000 people have been diagnosed with HPP in the U.S., however, the disease is often underdiagnosed due to a broad spectrum of symptoms that can mimic more common conditions.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.
To learn more, please visit www.biomarin.com.
About Alesta Therapeutics
Alesta Therapeutics is a clinical-stage biotechnology company committed to developing novel oral small-molecule therapies for underserved diseases. The company's lead asset, ALE1, is being developed for hypophosphatasia (HPP), a rare genetic disorder with significant unmet need.
For more information, visit www.alestatherapeutics.com.
Forward-Looking Statements
This press release contains forward-looking statements about, among other things, the proposed acquisition of ALE1, the lead clinical-stage asset, of Alesta Therapeutics (Alesta) by BioMarin Pharmaceutical Inc. (BioMarin) and the business prospects of BioMarin, including, without limitation, statements about: the anticipated occurrence, manner, funding and timing of the closing of the proposed acquisition; BioMarin's plans to update financial guidance; the potential impact of the acquisition on BioMarin's financial results and financial guidance; the prospective benefits of the proposed acquisition, including expectations that it will be a strong strategic fit for BioMarin and will meaningfully strengthen BioMarin's early-stage clinical pipeline; expectations regarding ALE1 and its ongoing development, including its potential to be a first-in-class oral therapy for the treatment of hypophosphatasia (HPP) and the potential benefits of ALE1 to patients with HPP around the world; BioMarin's plans to drive durable growth and strengthen its pipeline for the future; BioMarin's ability to compete in larger rare disease markets; BioMarin's expectations regarding unmet need and opportunities in HPP that may potentially be addressed by ALE1, including BioMarin's estimates regarding the prevalence of HPP; and other statements that are not historical facts.
These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: consummating the proposed acquisition in the anticipated timeframe, if at all; Alesta's ability to complete the contemplated spinout of non-ALE1 assets prior to closing of the proposed acquisition, if at all; the possibility that competing offers or acquisition proposals will be made; the possibility that various closing conditions for the transaction may not be satisfied or waived, including that a governmental entity may prohibit, delay, or refuse to grant approval for the consummation of the transaction (or only grant approval subject to adverse conditions or limitations); the difficulty of predicting the timing or outcome of regulatory approvals or actions, if any; the effects of the proposed acquisition (or the announcement thereof) on BioMarin's stock price and/or BioMarin's operating results; unknown or inestimable liabilities; the development, launch and commercialization of products and product candidates; BioMarin's ability to realize the anticipated benefits of the proposed acquisition, including the possibility that the expected benefits from the proposed acquisition will not be realized or will not be realized within the expected time period and that integration will not be successful or that such integration may be more difficult, time-consuming or costly than expected; the time-consuming and uncertain regulatory approval process for pharmaceutical product development; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to BioMarin's business operations and financial results; the sufficiency of BioMarin's cash flows and capital resources; BioMarin's ability to fund the acquisition; BioMarin's evaluation of the potential impact of the transaction on its financial results and financial guidance; BioMarin's ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the effects of the transaction on relationships with key third parties, including employees, customers, suppliers, other business partners or governmental entities; transaction costs; risks that the proposed acquisition disrupts current plans and operations; risks that the proposed transaction diverts management's attention from ongoing business operations; changes in Alesta's business during the period between announcement and closing of the proposed acquisition; any legal proceedings and/or regulatory actions that may be instituted related to the proposed acquisition; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
Investors interested in Medical stocks should always be looking to find the best-performing companies in the group. Is BioMarin Pharmaceutical (BMRN - Free Report) one of those stocks right now? A quick glance at the company's year-to-date performance in comparison to the rest of the Medical sector should help us answer this question.
BioMarin Pharmaceutical is one of 914 individual stocks in the Medical sector. Collectively, these companies sit at #6 in the Zacks Sector Rank. The Zacks Sector Rank considers 16 different groups, measuring the average Zacks Rank of the individual stocks within the sector to gauge the strength of each group.
The Zacks Rank is a proven model that highlights a variety of stocks with the right characteristics to outperform the market over the next one to three months. The system emphasizes earnings estimate revisions and favors companies with improving earnings outlooks. BioMarin Pharmaceutical is currently sporting a Zacks Rank of #2 (Buy).
Within the past quarter, the Zacks Consensus Estimate for BMRN's full-year earnings has moved 0.4% higher. This signals that analyst sentiment is improving and the stock's earnings outlook is more positive.
According to our latest data, BMRN has moved about 15.3% on a year-to-date basis. Meanwhile, the Medical sector has returned an average of 3% on a year-to-date basis. This shows that BioMarin Pharmaceutical is outperforming its peers so far this year.
Another stock in the Medical sector, Cardinal Health (CAH - Free Report) , has outperformed the sector so far this year. The stock's year-to-date return is 12.5%.
Over the past three months, Cardinal Health's consensus EPS estimate for the current year has increased 4.6%. The stock currently has a Zacks Rank #2 (Buy).
Breaking things down more, BioMarin Pharmaceutical is a member of the Medical - Biomedical and Genetics industry, which includes 439 individual companies and currently sits at #153 in the Zacks Industry Rank. On average, stocks in this group have gained 5.2% this year, meaning that BMRN is performing better in terms of year-to-date returns.
In contrast, Cardinal Health falls under the Medical - Dental Supplies industry. Currently, this industry has 14 stocks and is ranked #45. Since the beginning of the year, the industry has moved +4.6%.
Investors interested in the Medical sector may want to keep a close eye on BioMarin Pharmaceutical and Cardinal Health as they attempt to continue their solid performance.
Can BioMarin Stock Live Up to Wall Street’s High Expectations?BioMarin Pharmaceutical NASDAQ: BMRN outlined its growth strategy, integration plans for Amicus Therapeutics and pipeline priorities during a Canaccord discussion with Chief Financial Officer Brian Mueller.
Mueller described BioMarin as one of the largest rare-disease-focused biopharmaceutical companies, with more than 25 years of operating history, six first-in-disease medicines and nine approved therapies. The company operates in more than 80 countries and expects revenue approaching $4 billion this year, he said.
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3 Oversold Stocks with Big RSI Rebound PotentialThe company’s strategy centers on genetically defined rare diseases, where BioMarin seeks to identify the underlying genetic cause of a condition and develop precision medicines targeted at that cause. Mueller said that approach can support smaller clinical studies, regulatory incentives and strong engagement with patient advocacy communities.
Quarterly Performance and Amicus Integration BioMarin reported nearly $1 billion in second-quarter revenue, representing 20% year-over-year growth, Mueller said. The company raised total-revenue guidance to a range of $3.875 billion to $3.925 billion and raised non-GAAP earnings-per-share guidance to $4.90 to $5.10.
Are Gene Therapy Stocks The Market's Next Big Winners?During the quarter, BioMarin completed its acquisition of Amicus Therapeutics, adding two commercial rare-disease products: Galafold for Fabry disease and Pombiliti and Opfolda for Pompe disease. Mueller characterized the products as high-growth assets that can benefit from BioMarin’s larger global infrastructure.
BioMarin estimates peak revenue of $1.4 billion for Galafold and $1.2 billion for Pombiliti and Opfolda, with those opportunities expected in the mid- to late 2030s, respectively. The company also expects to realize synergies equal to roughly 50% of Amicus’ legacy operating-cost base, primarily through general and administrative expenses and other support functions.
Mueller said BioMarin is preserving and investing in the acquired sales and marketing organization rather than pursuing aggressive commercial cost reductions. “These products are in their high-growth phase,” he said, adding that maintaining customer-facing capabilities is important to sustaining growth.
BioMarin originally targeted a leverage ratio below 2.5 times within two years of the acquisition’s closing. Following its assessment of revenue opportunities, operating synergies and the combined company’s EBITDA profile, management now expects to reach that target by the middle of next year, roughly a year earlier than originally planned.
The company raised approximately $3.6 billion in debt for the transaction and also has a $600 million convertible note maturing next May, Mueller said. By 2030, BioMarin expects the Amicus business to operate at an approximately 60% operating margin.
Commercial Opportunities for Galafold and Pompe Franchise Mueller cited geographic expansion as a major growth driver for the acquired portfolio. Amicus had commercialized Galafold in roughly 40 markets and Pombiliti in approximately 15 markets, compared with BioMarin’s presence in more than 80 countries.
BioMarin does not expect to bring both medicines to every market where it operates, but Mueller said its on-the-ground commercial, market-access, medical and regulatory teams could accelerate international expansion in complex markets.
For Galafold, BioMarin sees an opportunity to improve diagnosis of Fabry disease, which Mueller said remains significantly underdiagnosed. The company plans to use efforts including AI-enabled patient identification, newborn screening and familial genetic cascade screening.
For Pombiliti and Opfolda, BioMarin’s strategy includes encouraging treatment switches from existing enzyme therapies. Mueller said there is an emerging body of real-world evidence suggesting improved outcomes with Pombiliti and Opfolda, while the efficacy of some existing therapies can wane over time.
Pipeline, Business Development and VOXZOGO BioMarin’s business-development focus remains centered on rare, genetically defined diseases. The company plans to prioritize opportunities that can use its internal manufacturing, development, regulatory, clinical and commercial infrastructure, Mueller said.
Management will continue to pursue early-stage collaborations and in-licensing opportunities, including a recently announced collaboration with the n-Lorem Foundation for a rare neurologic disease. As BioMarin reduces leverage, it may consider larger mid- to late-stage opportunities, though Mueller did not provide specifics on deal size or timing.
The company also raised revenue guidance for VOXZOGO to at least $1 billion after what Mueller described as strong first-half performance. VOXZOGO revenue grew 14% in the first quarter, and BioMarin added 20% new patients globally, he said.
VOXZOGO is approved for achondroplasia, and BioMarin has submitted a supplemental new drug application to the FDA for hypochondroplasia following positive Phase III results. If the filing is accepted, a potential approval and launch could occur next year, according to Mueller.
BioMarin estimates there are about 24,000 achondroplasia patients worldwide and approximately 14,000 hypochondroplasia patients. Mueller said diagnosis will be a key challenge in hypochondroplasia, but the company believes its established VOXZOGO presence in achondroplasia provides a foundation for the potential label expansion.
Separately, Mueller highlighted BMN 820, formerly DMX-200 at Amicus, as a potentially meaningful development asset. The small-molecule treatment is being studied for focal segmental glomerulosclerosis, or FSGS, a kidney disorder. BioMarin holds U.S. rights, estimates a U.S. population of roughly 30,000 patients and noted that the FDA agreed to proteinuria as the primary endpoint in the fully enrolled two-year study.
About BioMarin Pharmaceutical (NASDAQ:BMRN)BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company's commercial portfolio includes several approved therapies targeting inherited disorders.
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected].
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Value ScoreFinding good stocks at good prices, and discovering which companies are trading under their true value, are what value investors like to focus on. So, the Value Style Score takes into account ratios like P/E, PEG, Price/Sales, Price/Cash Flow, and a host of other multiples to highlight the most attractive and discounted stocks.
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Momentum ScoreMomentum traders and investors live by the saying "the trend is your friend." This investing style is all about taking advantage of upward or downward trends in a stock's price or earnings outlook. Employing factors like one-week price change and the monthly percentage change in earnings estimates, the Momentum Style Score can indicate favorable times to build a position in high-momentum stocks.
VGM ScoreIf you want a combination of all three Style Scores, then the VGM Score will be your friend. It rates each stock on their combined weighted styles, helping you find the companies with the most attractive value, best growth forecast, and most promising momentum. It's also one of the best indicators to use with the Zacks Rank.
How Style Scores Work with the Zacks Rank The Zacks Rank, which is a proprietary stock-rating model, employs earnings estimate revisions, or changes to a company's earnings expectations, to make building a winning portfolio easier.
It's highly successful, with #1 (Strong Buy) stocks producing an unmatched +23.94% average annual return since 1988. That's more than double the S&P 500. But because of the large number of stocks we rate, there are over 200 companies with a Strong Buy rank, plus another 600 with a #2 (Buy) rank, on any given day.
This totals more than 800 top-rated stocks, and it can be overwhelming to try and pick the best stocks for you and your portfolio.
That's where the Style Scores come in.
You want to make sure you're buying stocks with the highest likelihood of success, and to do that, you'll need to pick stocks with a Zacks Rank #1 or #2 that also have Style Scores of A or B. If you like a stock that only has a #3 (Hold) rank, it should also have Scores of A or B to guarantee as much upside potential as possible.
The direction of a stock's earnings estimate revisions should always be a key factor when choosing which stocks to buy, since the Scores were created to work together with the Zacks Rank.
Here's an example: a stock with a #4 (Sell) or #5 (Strong Sell) rating, even one with Style Scores of A and B, still has a downward-trending earnings outlook, and a bigger chance its share price will decrease too.
Thus, the more stocks you own with a #1 or #2 Rank and Scores of A or B, the better.
Stock to Watch: BioMarin Pharmaceutical (BMRN - Free Report) San Rafael, CA-based BioMarin Pharmaceutical Inc. focuses on the development and commercialization of treatments for life-threatening severe medical conditions, mainly for children.
BMRN is a #2 (Buy) on the Zacks Rank, with a VGM Score of B.
It also boasts a Value Style Score of B thanks to attractive valuation metrics like a forward P/E ratio of 14.02; value investors should take notice.
Two analysts revised their earnings estimate upwards in the last 60 days for fiscal 2026. The Zacks Consensus Estimate has increased $0.01 to $4.96 per share. BMRN boasts an average earnings surprise of +67.5%.
With a solid Zacks Rank and top-tier Value and VGM Style Scores, BMRN should be on investors' short list.
BioMarin Pharmaceutical (BMRN - Free Report) closed the last trading session at $69.55, gaining 18.6% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $89.16 indicates a 28.2% upside potential.
The mean estimate comprises 25 short-term price targets with a standard deviation of $20.61. While the lowest estimate of $60.00 indicates a 13.7% decline from the current price level, the most optimistic analyst expects the stock to surge 78.3% to reach $124.00. It's very important to note the standard deviation here, as it helps understand the variability of the estimates. The smaller the standard deviation, the greater the agreement among analysts.
While the consensus price target is highly sought after by investors, the ability and unbiasedness of analysts in setting price targets have long been questionable. And investors making investment decisions solely based on this tool would arguably do themselves a disservice.
But, for BMRN, an impressive average price target is not the only indicator of a potential upside. Strong agreement among analysts about the company's ability to report better earnings than they predicted earlier strengthens this view. While a positive trend in earnings estimate revisions doesn't gauge how much a stock could gain, it has proven to be powerful in predicting an upside.
Price, Consensus and EPS Surprise
Here's What You May Not Know About Analysts' Price TargetsAccording to researchers at several universities across the globe, a price target is one of many pieces of information about a stock that misleads investors far more often than it guides. In fact, empirical research shows that price targets set by several analysts, irrespective of the extent of agreement, rarely indicate where the price of a stock could actually be heading.
While Wall Street analysts have deep knowledge of a company's fundamentals and the sensitivity of its business to economic and industry issues, many of them tend to set overly optimistic price targets. Are you wondering why?
They usually do that to drum up interest in shares of companies that their firms either have existing business relationships with or are looking to be associated with. In other words, business incentives of firms covering a stock often result in inflated price targets set by analysts.
However, a tight clustering of price targets, which is represented by a low standard deviation, indicates that analysts have a high degree of agreement about the direction and magnitude of a stock's price movement. While that doesn't necessarily mean the stock will hit the average price target, it could be a good starting point for further research aimed at identifying the potential fundamental driving forces.
That said, while investors should not entirely ignore price targets, making an investment decision solely based on them could lead to disappointing ROI. So, price targets should always be treated with a high degree of skepticism.
Why BMRN Could Witness a Solid UpsideAnalysts' growing optimism over the company's earnings prospects, as indicated by strong agreement among them in revising EPS estimates higher, could be a legitimate reason to expect an upside in the stock. That's because empirical research shows a strong correlation between trends in earnings estimate revisions and near-term stock price movements.
Over the last 30 days, the Zacks Consensus Estimate for the current year has increased 0.4%, as two estimates have moved higher compared to no negative revision.
Moreover, BMRN currently has a Zacks Rank #2 (Buy), which means it is in the top 20% of more than 4,000 stocks that we rank based on four factors related to earnings estimates. Given an impressive externally-audited track record, this is a more conclusive indication of the stock's potential upside in the near term. You can see the complete list of today's Zacks Rank #1 (Strong Buy) stocks here >>>> .
Therefore, while the consensus price target may not be a reliable indicator of how much BMRN could gain, the direction of price movement it implies does appear to be a good guide.
Resources Investor Relations Journalists Agencies Client Login Send a Release News Products Contact , /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced management will present at the Canaccord Genuity 46th Annual Growth Conference on Wednesday, August 12, 2026, at 8:00AM ET in Boston.
An audio webcast of the presentation will be available live. The webcast can be accessed at: https://investors.biomarin.com/. An archived version of the remarks will also be available through the Company's website for a limited time following the conference.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Key Takeaways BioMarin posted a Q2 revenue beat, led by Voxzogo, Amicus-acquired products and stronger Palynziq demand.Voxzogo sales rose 14%, while Galafold added $106 million following the Amicus acquisition.BMRN raised 2026 revenues, Voxzogo and adjusted EPS guidance after a strong second-quarter performance. BioMarin Pharmaceutical (BMRN - Free Report) reported second-quarter 2026 adjusted earnings of $1.20 per share, which beat the Zacks Consensus Estimate of 96 cents. However, the bottom line declined 16.7% year over year from $1.44.
Total revenues were $989.7 million, up 20% year over year, beating the Zacks Consensus Estimate of $922 million. Growth was driven by new Amicus-acquired products, Voxzogo patient additions and stronger Palynziq demand.
BMRN Stock MovementShares of BioMarin were up 4.4% in after-market trading on Thursday following the earnings announcement. The positive stock reaction came as both earnings and revenues topped expectations and management raised key elements of its 2026 financial guidance.
Year to date, BMRN stock has gained 3.4% compared with the industry’s 3.6% growth.
Image Source: Zacks Investment Research
More on BioMarin's Q2 EarningsNet product revenues totaled nearly $984.4 million, up about 21% year over year, driven by higher revenues from the company’s Metabolic Conditions drugs, as well as Voxzogo.
Voxzogo generated $253 million in sales, up 14% year over year from $221 million. Sales also beat the Zacks Consensus Estimate of $238.2 million. The number of children treated globally increased more than 20% year over year.
BioMarin said about 90% of U.S. children treated with Voxzogo remained on therapy through the end of July after a competing product entered the market. More than half of new U.S. patient starts during the quarter were children under two years of age.
BMRN's Metabolic Conditions PerformanceMetabolic Conditions revenues totaled $695 million, up 25% year over year. The portfolio now comprises seven therapies, including Galafold and Pombiliti-Opfolda, which were added following the Amicus acquisition.
Palynziq sales jumped 27% to $135 million, topping the Zacks Consensus Estimate of $112.7 million. Naglazyme revenues rose 5% to $135 million, which also came above the consensus estimate of $125.9 million. Brineura sales increased 4% to $51 million.
Vimizim revenues fell 10% to $194 million, reflecting the timing of large government orders outside the United States. Sales missed the Zacks Consensus Estimate of $205.8 million. Aldurazyme sales declined 21% to $44 million due to timing of order fulfillment to Sanofi (SNY - Free Report) .
Galafold contributed $106 million following the Amicus acquisition. BioMarin said the therapy maintained broad-based patient growth, supported by increased diagnosis and patient identification. The Sanofi-related Aldurazyme order timing was a separate headwind within the portfolio.
BioMarin signed a collaboration agreement with Sanofi’s subsidiary, Genzyme, for Aldurazyme. SNY, through Genzyme, is BMRN’s sole customer for Aldurazyme. The Sanofi subsidiary is responsible for marketing and selling Aldurazyme to third parties.
BioMarin's Other RevenuesKuvan generated $24 million in second-quarter sales, down 11% from $27 million a year earlier.
Roctavian revenues were $12 million, up 33% year over year from $9 million.
Royalty and other revenues were $5.3 million compared with $12.4 million in the year-ago quarter.
BMRN Raises 2026 Financial OutlookBioMarin raised its 2026 total revenue guidance to $3.875-$3.925 billion from $3.825-$3.925 billion. Metabolic Conditions revenue guidance was maintained at $2.725-$2.775 billion.
Voxzogo revenue guidance was increased to $1-$1.05 billion from $975-$1.025 billion. Other revenues are expected to be in the range of $100-$125 million in 2026, unchanged from the previous guidance. Adjusted earnings guidance was raised to $4.90-$5.10 per share from $4.85-$5.05.
BioMarin's Key UpdatesBioMarin submitted a supplemental new drug application (NDA) to the FDA seeking approval of Voxzogo for hypochondroplasia after the phase III CANOPY-HCH-3 study met its primary endpoint. The company expects to provide an update on the application status with its third-quarter earnings update.
The FDA also accepted BioMarin's supplemental NDA for full approval of Voxzogo in children with achondroplasia, with a Feb. 28, 2027, target action date. Meanwhile, the company discontinued BMN 401 after the ENERGY 3 study failed to meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency. A data update from the phase II/III study of BMN 333 for achondroplasia is expected in 2027.
In the second quarter, the European Commission approved Palynziq for adolescents aged 12 years and older with phenylketonuria (PKU). The label expansion broadens access to Palynziq, which enables patients with PKU to achieve physiologic phenylalanine levels while reducing dietary restrictions, regardless of disease severity.
Our Take on BMRN’s Q2 ResultsBioMarin's quarter showed broad commercial momentum, with Voxzogo growth and the newly acquired Galafold contribution supporting a strong revenue beat. Palynziq and Naglazyme also exceeded expectations, while Vimizim and Aldurazyme faced order-timing pressure.
The raised outlook adds to the positive read-through from the quarter, though product-level volatility remains evident. In particular, Aldurazyme's decline reflected order fulfillment timing to Sanofi, making SNY-related ordering an important factor to watch alongside competitive dynamics in Voxzogo.
BMRN’s Zacks Rank & Stocks to ConsiderBioMarin currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Harmony Biosciences (HRMY - Free Report) and Liquidia Corporation (LQDA - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Harmony Biosciences’ 2026 earnings per share have risen from $3.20 to $3.33, while estimates for 2027 have increased from $3.64 to $3.92 during the same time. HRMY shares have gained 3.5% year to date.
Harmony Biosciences’ earnings missed estimates in three of the trailing four quarters and beat on the remaining occasion, delivering an average negative surprise of 13.97%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $2.97 to $3.02, while estimates for 2027 have increased from $4.81 to $5.31 during the same time. LQDA shares have surged 159.3% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.
BioMarin Pharmaceutical Inc. (BMRN) Q2 2026 Earnings Call August 6, 2026 4:30 PM EDT
Company Participants
Traci McCarty - Group Vice President
Alexander Hardy - President, CEO & Director
Cristin Hubbard - Executive VP & Chief Commercial Officer
Gregory Friberg - Executive VP and Chief Research & Development Officer
Brian Mueller - CFO & Executive VP of Finance
Conference Call Participants
Christopher Raymond - Raymond James & Associates, Inc., Research Division
Cory Kasimov - Evercore ISI Institutional Equities, Research Division
Jessica Fye - JPMorgan Chase & Co, Research Division
Tommie Reerink - Goldman Sachs Group, Inc., Research Division
Philip Nadeau - TD Cowen, Research Division
Eliana Merle - Barclays Bank PLC, Research Division
Mohit Bansal - Wells Fargo Securities, LLC, Research Division
Phoebe Tan - Jefferies LLC, Research Division
Paul Matteis - Stifel, Nicolaus & Company, Incorporated, Research Division
Sean Laaman - Morgan Stanley, Research Division
Alexandria Hammond - Wolfe Research, LLC
Presentation
Operator
Good afternoon, and welcome, everyone, to the BioMarin Pharmaceutical Second Quarter 2026 Conference Call. Today's conference is being recorded. [Operator Instructions]
At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations.
Traci McCarty
Group Vice President
Thank you, operator, and thank you all for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.
In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP
Can BioMarin Stock Live Up to Wall Street’s High Expectations?BioMarin Pharmaceutical NASDAQ: BMRN reported second-quarter 2026 revenue of nearly $1 billion, up 20% from a year earlier, as growth across its rare-disease portfolio and the recently completed Amicus acquisition supported results. The company raised its full-year revenue, VOXZOGO revenue and non-GAAP diluted earnings-per-share guidance, though it did not provide updated ranges during the call.
Chief Executive Officer Alexander Hardy said the quarter combined strong commercial execution with the close and integration of Amicus. BioMarin expects the acquired GALAFOLD and POMBILITI and OPFOLDA therapies to expand its growth profile through the mid-2030s, supported by international expansion, patient identification efforts and planned cost savings.
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Amicus integration and growth outlook 3 Oversold Stocks with Big RSI Rebound PotentialBioMarin projected peak revenue of $1.4 billion for GALAFOLD, a treatment for Fabry disease, and $1.2 billion for POMBILITI and OPFOLDA, a combination therapy for Pompe disease. The company expects GALAFOLD revenue to grow at an approximately 10% compound annual rate from 2027 through 2032, while it expects POMBILITI and OPFOLDA to grow at a rate of at least 20% over the same period.
On a pro forma basis, GALAFOLD revenue increased about 10% year over year in the second quarter, while POMBILITI and OPFOLDA revenue rose more than 65%, according to Chief Commercial Officer Cristin Hubbard. GALAFOLD’s growth was driven by patient additions in established and newer markets, while POMBILITI and OPFOLDA added patients in the U.S. and recently launched geographies.
Are Gene Therapy Stocks The Market's Next Big Winners?The company expects approximately $220 million in annual run-rate cost synergies to be fully realized in 2028, representing about a 50% reduction from Amicus’ 2025 non-GAAP operating expenses of $432 million. More than 70% of the expected savings are expected to come from general and administrative expenses, with the remaining savings primarily from research and development.
Chief Financial Officer Brian Mueller said the acquisition is still expected to be modestly dilutive in calendar 2026, though it is “close to breakeven.” BioMarin expects substantial earnings accretion to begin in 2027, with roughly half to slightly more than half of the planned synergies expected to be realized next year. The company also expects to reduce leverage about one year earlier than previously communicated.
Mueller said acquisition-related debt is expected to generate annualized interest expense of roughly $200 million, or about $50 million per quarter, based on current rates. That expense is included in non-GAAP results. Interest income is also expected to decline in the near term following the use of cash and investments to fund the acquisition.
VOXZOGO growth continues amid competition VOXZOGO revenue grew 14% year over year in the second quarter, with double-digit growth in both the U.S. and international markets. The number of children receiving the treatment increased more than 20% globally from a year earlier, and approximately three-quarters of VOXZOGO revenue came from outside the U.S.
BioMarin said it raised its full-year VOXZOGO outlook following first-half performance and expectations for the remainder of 2026. Mueller said one international pricing negotiation closed with a favorable outcome, while another remained in process after initial setbacks. Growth in both the U.S. and global markets also contributed to the improved outlook.
The company is managing competition in the U.S. achondroplasia market following a competitor’s February launch. Hubbard said that approximately 90% of U.S. children using VOXZOGO had remained on treatment as of the end of July, based on the company’s available information. Hardy said BioMarin had observed approximately 10% of U.S. VOXZOGO patients switching, or fewer than 100 patients over roughly six months.
Hubbard said patients who switched were primarily seeking less frequent dosing or responding to injection fatigue. BioMarin said more than half of its new U.S. patient starts during the quarter were in children ages two and younger, an age group for which it said VOXZOGO remains the only approved treatment. Chief Research and Development Officer Greg Friberg estimated there are about 150 U.S. births annually involving infants with achondroplasia.
BioMarin expects both patient additions and ordering patterns to lift VOXZOGO revenue in the second half compared with the first half. The company said VOXZOGO is on track to become its first $1 billion product.
Pipeline and commercial updates BioMarin submitted a supplemental new drug application for VOXZOGO in hypochondroplasia, following pivotal data reported during the quarter. Friberg said the company reduced the time from database lock to filing to 79 days through parallel work processes and technology-enabled efforts. Full Phase III results are scheduled for presentation at the ESPE meeting in September, and BioMarin plans to provide an update on the filing with third-quarter results.
The company estimates a global addressable hypochondroplasia population of approximately 14,000 patients. BioMarin said it is pursuing physician education, digital campaigns, genetic testing and other patient-identification initiatives ahead of a potential launch.
The Amicus transaction also added BMN 820, formerly DMX-200, to BioMarin’s pipeline. The oral CCR2 inhibitor is in Phase III development for focal segmental glomerulosclerosis, or FSGS. BioMarin holds exclusive U.S. commercialization rights, while partner Dimerix is responsible for operating the Phase III ACTION 3 study. The company expects Phase III data in 2028.
BioMarin’s Metabolic Conditions business, formerly called Enzyme Therapies, generated $695 million in second-quarter revenue, up 25% year over year including the acquired Amicus products. PALYNZIQ revenue increased 27%, aided by patient demand and U.S. ordering timing. The company also recently received European approval to expand PALYNZIQ’s label to adolescents ages 12 and older with PKU.
For the second half, Mueller said third-quarter revenue should be slightly above the second quarter, reflecting a full quarter of Amicus contributions. He expects the fourth quarter to be the company’s strongest of 2026, with ordering dynamics in select markets accounting for well over half of projected second-half revenue.
About BioMarin Pharmaceutical (NASDAQ:BMRN)BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company's commercial portfolio includes several approved therapies targeting inherited disorders.
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BioMarin Pharmaceutical (BMRN - Free Report) reported $989.71 million in revenue for the quarter ended June 2026, representing a year-over-year increase of 19.9%. EPS of $1.20 for the same period compares to $1.44 a year ago.
The reported revenue represents a surprise of +7.31% over the Zacks Consensus Estimate of $922.33 million. With the consensus EPS estimate being $0.96, the EPS surprise was +25%.
While investors closely watch year-over-year changes in headline numbers -- revenue and earnings -- and how they compare to Wall Street expectations to determine their next course of action, some key metrics always provide a better insight into a company's underlying performance.
As these metrics influence top- and bottom-line performance, comparing them to the year-ago numbers and what analysts estimated helps investors project a stock's price performance more accurately.
Here is how BioMarin performed in the just reported quarter in terms of the metrics most widely monitored and projected by Wall Street analysts:
Revenues- Net product revenues: $984.39 million versus the eight-analyst average estimate of $914.12 million. The reported number represents a year-over-year change of +21.1%.Revenues- Royalty and other revenues: $5.32 million compared to the $8.21 million average estimate based on eight analysts. The reported number represents a change of -57.2% year over year.Revenues- Net Product Revenues- NAGLAZYME: $135 million compared to the $125.91 million average estimate based on seven analysts. The reported number represents a change of +4.7% year over year.Revenues- Net Product Revenues- VIMIZIM: $194 million compared to the $205.82 million average estimate based on seven analysts. The reported number represents a change of -9.8% year over year.Revenues- Net Product Revenues- PALYNZIQ: $135 million versus $112.75 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +27.4% change.Revenues- Net Product Revenues- VOXZOGO: $253 million versus the seven-analyst average estimate of $238.2 million. The reported number represents a year-over-year change of +14.5%.Revenues- Net Product Revenues- KUVAN: $24 million versus the seven-analyst average estimate of $17.25 million. The reported number represents a year-over-year change of -11.1%.Revenues- Net Product Revenues- ALDURAZYME: $44 million versus the seven-analyst average estimate of $52.41 million. The reported number represents a year-over-year change of -21.4%.Revenues- Net Product Revenues- BRINEURA: $51 million versus $50.06 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +4.1% change.View all Key Company Metrics for BioMarin here>>>
Shares of BioMarin have returned +0.3% over the past month versus the Zacks S&P 500 composite's +3.3% change. The stock currently has a Zacks Rank #3 (Hold), indicating that it could perform in line with the broader market in the near term.
BioMarin Pharmaceutical (BMRN - Free Report) came out with quarterly earnings of $1.2 per share, beating the Zacks Consensus Estimate of $0.96 per share. This compares to earnings of $1.44 per share a year ago. These figures are adjusted for non-recurring items.
This quarterly report represents an earnings surprise of +25.00%. A quarter ago, it was expected that this rare disease biopharmaceutical would post earnings of $0.94 per share when it actually produced earnings of $0.76, delivering a surprise of -19.15%.
Over the last four quarters, the company has surpassed consensus EPS estimates three times.
BioMarin, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $989.71 million for the quarter ended June 2026, surpassing the Zacks Consensus Estimate by 7.31%. This compares to year-ago revenues of $825.41 million. The company has topped consensus revenue estimates three times over the last four quarters.
The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.
BioMarin shares have added about 0.8% since the beginning of the year versus the S&P 500's gain of 12.8%.
What's Next for BioMarin?While BioMarin has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?
There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.
Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.
Ahead of this earnings release, the estimate revisions trend for BioMarin was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.
It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $1.49 on $1.04 billion in revenues for the coming quarter and $4.95 on $3.86 billion in revenues for the current fiscal year.
Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the top 44% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.
BioXcel Therapeutics, Inc. (BTAI - Free Report) , another stock in the same industry, has yet to report results for the quarter ended June 2026.
This company is expected to post quarterly loss of $0.51 per share in its upcoming report, which represents a year-over-year change of +79.2%. The consensus EPS estimate for the quarter has remained unchanged over the last 30 days.
BioXcel Therapeutics, Inc.'s revenues are expected to be $0.34 million, up 183.3% from the year-ago quarter.
Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million
Stronger Growth Expectations Drive Increased Guidance for Full-year 2026 Total Revenues, VOXZOGO®, and Non-GAAP Diluted Earnings Per Share (EPS)
Addition of GALAFOLD® and POMBILITI® + OPFOLDA®, with Cost Synergies, Expected to Accelerate Revenue Growth, Non-GAAP Diluted EPS Accretion, Non-GAAP Operating Margin Expansion, and Operating Cash Flow through the Mid-2030s
Conference Call and Webcast Scheduled Today at 4:30 p.m. ET
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the second quarter ended June 30, 2026.
"This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026. Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations." Mr. Hardy added, "With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world."
2026 Business and Pipeline Highlights
Innovation
BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update. In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span. The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September. In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score (SDS) improving 0.72 over the three years. Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia. The Phase 2/3 study is enrolling, with a data update expected in 2027. In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027. In the second quarter, the European Commission approved PALYNZIQ® for adolescents 12 years and older with phenylketonuria (PKU). PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028. BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development. The company expects to provide a program update by year-end. Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications. In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. ReNU syndrome has an expected global population of approximately 100,000. Growth
BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s. BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities. Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ. The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially. Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion. The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter. In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter. Value Commitment
As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. Synergies reflect a reduction of Amicus' legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth. GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030. The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company. Second Quarter 2026 Financial Highlights
Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ. These increases were partially offset by lower VIMIZIM® revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME® sales volume due to the timing of order fulfillment to Sanofi. GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025. The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up. Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above. Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025. The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above. Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)
Three Months Ended
June 30,
Six Months Ended
June 30,
2026
2025
% Change
2026
2025
% Change
Total Revenues
$990
$825
20 %
$1,756
$1,571
12 %
Net Product Revenues by Product:
VOXZOGO
$253
$221
14 %
$472
$435
9 %
Metabolic Conditions:
VIMIZIM
$194
$215
(10) %
$405
$404
— %
NAGLAZYME®
135
129
5 %
265
243
9 %
PALYNZIQ
135
106
27 %
225
199
13 %
GALAFOLD
106
—
NM
106
—
NM
BRINEURA®
51
49
4 %
98
89
10 %
ALDURAZYME
44
56
(21) %
80
105
(24) %
POMBILITI + OPFOLDA
30
—
NM
30
—
NM
Total Metabolic Conditions Revenue
$695
$555
25 %
$1,209
$1,040
16 %
KUVAN®
$24
$27
(11) %
$48
$52
(8) %
ROCTAVIAN®(1)
$12
$9
33 %
$14
$20
(30) %
GAAP Net Income
$45
$241
(81) %
$150
$426
(65) %
Non-GAAP Income (2)
$236
$282
(16) %
$385
$502
(23) %
GAAP Operating Margin % (3)
11.2 %
33.5 %
13.7 %
31.9 %
Non-GAAP Operating Margin % (2)
36.4 %
39.9 %
31.0 %
37.9 %
GAAP Diluted EPS
$0.23
$1.23
(81) %
$0.77
$2.19
(65) %
Non-GAAP Diluted EPS (2)
$1.20
$1.44
(17) %
$1.96
$2.57
(24) %
NM
Percentage change is not meaningful for products acquired from Amicus on April 27, 2026.
(1)
In 2026, the company announced that it will no longer market ROCTAVIAN.
(2)
Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin percentage and Non-GAAP Diluted EPS along with the related reconciliations to the comparable information reported under U.S. GAAP.
(3)
GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues.
Forward-Looking Non-GAAP Financial Information
BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.
Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)
Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO. Guidance reflects post-close contributions from Amicus beginning April 27, 2026. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Based on current rates, interest expense associated with the financing is estimated at approximately $200 million on an annualized basis, with Term Loans and Senior Notes scheduled to mature after 2030. Item
Provided on May 4, 2026
Updated August 6, 2026
Midpoint Growth
(Y/Y)
Total Revenues
$3,825
to
$3,925
$3,875
to
$3,925
21 %
Metabolic Conditions
$2,725
to
$2,775
Unchanged
31 %
VOXZOGO
$975
to
$1,025
$1,000
to
$1,050
11 %
Other Revenues(1)
$100
to
$125
Unchanged
Non-GAAP Diluted EPS (2)(3)(4)
$4.85
to
$5.05
$4.90
to
$5.10
59 %
(1)
Other Revenues includes KUVAN, ROCTAVIAN, and royalties.
(2)
Refer to Non-GAAP Information beginning on page 10 of this press release for definition of Non-GAAP Diluted EPS.
(3)
Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.
(4)
Non-GAAP Diluted EPS guidance assumes a combined company tax rate of 22%.
BioMarin will host a conference call and webcast to discuss second quarter 2026 financial results today, Thursday, August 6, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.
U.S./Canada Dial-in Number: 800-715-9871
Replay Dial-in Number: 800-770-2030
International Dial-in Number: 646-307-1963
Replay International Dial-in Number: 609-800-9909
Conference ID: 3551298
Conference ID: 3551298
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Diluted EPS, Non-GAAP Operating Margin, gross leverage, operating cash flow and revenue compound annual growth rate (CAGR) for, in certain instances, the full-year 2026, fourth quarter and second half of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Metabolic Conditions portfolio, including PALYNZIQ, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA, including BioMarin's plans and expectations to accelerate growth through mid-2030s; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products.
These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to realize the anticipated benefits of any acquisitions; BioMarin's ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF INCOME
Three and Six Months Ended June 30, 2026 and 2025
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
Three Months Ended
June 30,
Six Months Ended
June 30,
2026
2025
2026
2025
REVENUES:
Net product revenues
$ 984,393
$ 812,982
$ 1,744,471
$ 1,547,626
Royalty and other revenues
5,315
12,428
11,445
22,929
Total revenues
989,708
825,410
1,755,916
1,570,555
OPERATING EXPENSES:
Cost of sales
202,795
150,090
397,794
301,648
Research and development
206,952
161,308
385,748
320,039
Selling, general and administrative
395,549
232,279
653,839
438,395
Intangible asset amortization
73,492
4,846
77,975
9,693
Total operating expenses
878,788
548,523
1,515,356
1,069,775
INCOME FROM OPERATIONS
110,920
276,887
240,560
500,780
Interest income
10,480
18,827
33,040
37,840
Interest expense
(63,295)
(2,679)
(78,253)
(5,542)
Other income, net
3,279
4,833
7,240
2,879
INCOME BEFORE INCOME TAXES
61,384
297,868
202,587
535,957
Provision for income taxes
16,622
57,336
52,298
109,739
NET INCOME
$ 44,762
$ 240,532
$ 150,289
$ 426,218
EARNINGS PER SHARE, BASIC
$ 0.23
$ 1.25
$ 0.78
$ 2.23
EARNINGS PER SHARE, DILUTED
$ 0.23
$ 1.23
$ 0.77
$ 2.19
Weighted average common shares outstanding, basic
193,423
191,907
192,959
191,440
Weighted average common shares outstanding, diluted
194,467
197,091
194,147
196,643
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
June 30, 2026 and December 31, 2025
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
June 30, 2026
December 31, 2025
ASSETS
Current assets:
Cash and cash equivalents
$ 874,005
$ 1,311,679
Short-term investments
—
248,930
Accounts receivable, net
1,061,047
908,214
Inventory
1,782,524
1,298,883
Other current assets
254,047
185,784
Total current assets
3,971,623
3,953,490
Noncurrent assets:
Long-term investments
—
492,242
Property, plant and equipment, net
989,994
952,508
Intangible assets, net
4,879,367
213,837
Goodwill
655,745
196,199
Deferred tax assets
888,575
1,508,697
Other assets
338,138
277,049
Total assets
$ 11,723,442
$ 7,594,022
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable and accrued liabilities
$ 1,005,793
$ 759,031
Current portion of long-term debt, net
658,203
—
Total current liabilities
1,663,996
759,031
Noncurrent liabilities:
Long-term debt, net
3,527,939
597,176
Other long-term liabilities
209,815
150,816
Total liabilities
5,401,750
1,507,023
Stockholders' equity:
Common stock, $0.001 par value: 500,000,000 shares authorized; 193,535,556 and 192,300,101 shares issued and outstanding, respectively
194
192
Additional paid-in capital
6,037,019
5,956,582
Company common stock held by the Nonqualified Deferred Compensation Plan
(11,233)
(10,508)
Accumulated other comprehensive income (loss)
(8,783)
(13,473)
Retained earnings
304,495
154,206
Total stockholders' equity
6,321,692
6,086,999
Total liabilities and stockholders' equity
$ 11,723,442
$ 7,594,022
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
Six Months Ended June 30, 2026 and 2025
(In thousands of U.S. dollars)
(Unaudited)
Six Months Ended June 30,
2026
2025
CASH FLOWS FROM OPERATING ACTIVITIES:
Net income
$ 150,289
$ 426,218
Adjustments to reconcile net income to net cash provided by operating activities:
Depreciation and amortization
114,049
40,632
Non-cash interest expense
27,912
1,320
Stock-based compensation
119,152
85,231
Impairment of assets
—
2,967
Deferred income taxes
2,261
61,771
Unrealized foreign exchange gains
(4,046)
(5,306)
Other
(5,534)
(4,633)
Changes in operating assets and liabilities, net of effects of business acquired:
Accounts receivable, net
(46,005)
(156,124)
Inventory
12,334
(72,462)
Other current assets
(23,589)
(15,092)
Other assets
9,005
(13,505)
Accounts payable and accrued liabilities
26,265
3,111
Other long-term liabilities
6,667
5,537
Net cash provided by operating activities
388,760
359,665
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property, plant and equipment
(49,739)
(33,869)
Maturities and sales of investments
767,277
195,738
Purchases of investments
(25,792)
(202,433)
Purchase of intangible assets
(5,433)
(266)
Acquisition of Amicus, net of cash acquired
(5,067,630)
—
Other
4,966
—
Net cash used in investing activities
(4,376,351)
(40,830)
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from exercises of awards under equity incentive plans
6,655
7,707
Taxes paid related to net share settlement of equity awards
(39,504)
(51,089)
Proceeds from borrowings
3,650,000
—
Payments of debt issuance costs
(65,604)
—
Net cash provided by (used in) financing activities
3,551,547
(43,382)
Effect of exchange rate changes on cash
(1,630)
(4,479)
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS
(437,674)
270,974
Cash and cash equivalents:
Beginning of period
$ 1,311,679
$ 942,842
End of period
$ 874,005
$ 1,213,816
Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Cost of Sales (COS), Non-GAAP R&D expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans or convertible debt in periods when they are dilutive under Non-GAAP. Projected Gross Leverage is defined by the company as undiscounted debt (total debt excluding unamortized discount and deferred offering costs) as of the balance sheet date divided by 4 quarter projected Non-GAAP Adjusted EBITDA. Non-GAAP Adjusted EBITDA is defined by the company as GAAP Income (or Loss) from Operations excluding the impact of depreciation, amortization and stock-based compensation expense. Non-GAAP synergies is defined by the company as Amicus' legacy labor and external spend cost reductions, excluding the impact of stock-based compensation.
BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business.
Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.
The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:
Reconciliation of GAAP Reported Information to Non-GAAP Information (1)
(In millions of U.S. dollars, except per share data)
(unaudited)
Three Months Ended
June 30,
Six Months Ended
June 30,
2026
2025
2026
2025
GAAP Reported Net Income
$ 45
$ 241
$ 150
$ 426
Adjustments
Stock-based compensation expense - COS
5
4
9
6
Stock-based compensation expense - R&D
16
14
28
26
Stock-based compensation expense - SG&A(2)
55
30
83
53
Amortization of intangible assets
73
5
78
10
Amortization of acquired inventory step-up(3)
12
—
12
—
Acquisition-related costs(3)
84
—
84
—
Severance costs (4)
3
—
12
—
Loss on investments (5)
—
—
—
3
Income tax effect of adjustments
(57)
(11)
(70)
(22)
Non-GAAP Income
$ 236
$ 282
$ 385
$ 502
Three Months Ended
June 30,
2026
2025
COS
R&D
SG&A
COS
R&D
SG&A
GAAP expenses
$ 203
$ 207
$ 396
$ 150
$ 161
$ 232
Adjustments
Stock-based compensation expense(2)
(5)
(16)
(55)
(4)
(14)
(30)
Amortization of acquired inventory step-up(3)
(12)
—
—
—
—
—
Acquisition-related costs (3)
—
—
(84)
—
—
—
Severance costs (4)
—
—
(3)
—
—
—
Non-GAAP expenses
$ 186
$ 191
$ 253
$ 146
$ 147
$ 203
Six Months Ended
June 30,
2026
2025
COS
R&D
SG&A
COS
R&D
SG&A
GAAP expenses
$ 398
$ 386
$ 654
$ 302
$ 320
$ 438
Adjustments
Stock-based compensation expense(2)
(9)
(28)
(83)
(6)
(26)
(53)
Amortization of acquired inventory step-up(3)
(12)
—
—
—
—
—
Acquisition-related costs (3)
—
—
(84)
—
—
—
Severance costs (4)
—
—
(12)
—
—
—
Non-GAAP expenses
$ 378
$ 358
$ 475
$ 295
$ 294
$ 385
Three Months Ended
June 30,
Six Months Ended
June 30,
2026
Percent
of GAAP
Total
Revenue
2025
Percent
of GAAP
Total
Revenue
2026
Percent
of GAAP
Total
Revenue
2025
Percent
of GAAP
Total
Revenue
GAAP Income from Operations
$ 111
11.2 %
$ 277
33.5 %
$ 241
13.7 %
$ 501
31.9 %
Adjustments
Stock-based compensation expense(2)
76
7.7
48
5.8
120
6.8
85
5.4
Amortization of intangible assets
73
7.4
5
0.6
78
4.4
10
0.6
Amortization of acquired inventory step-up(3)
12
1.2
—
—
12
0.7
—
—
Acquisition-related costs (3)
84
8.5
—
—
84
4.8
—
—
Severance costs (4)
3
0.3
—
—
12
0.7
—
—
Non-GAAP Income from Operations
$ 360
36.4 %
$ 329
39.9 %
$ 545
31.0 %
$ 596
37.9 %
Three Months Ended
June 30,
Six Months Ended
June 30,
2026
2025
2026
2025
GAAP Diluted EPS
$ 0.23
$ 1.23
$ 0.77
$ 2.19
Adjustments
Stock-based compensation expense(2)
$ 0.38
$ 0.24
0.60
0.43
Amortization of intangible assets
$ 0.37
$ 0.03
0.39
0.05
Amortization of acquired inventory step-up(3)
$ 0.06
$ —
0.06
—
Acquisition-related costs(3)
$ 0.42
$ —
0.42
—
Severance costs (4)
$ 0.02
$ —
0.06
—
Loss on investments (5)
$ —
$ —
—
0.02
Income tax effect of adjustments
$ (0.29)
$ (0.06)
(0.35)
(0.11)
Non-GAAP Diluted EPS
$ 1.20
$ 1.44
$ 1.96
$ 2.57
Three Months Ended
June 30,
Six Months Ended
June 30,
2026
2025
2026
2025
GAAP Weighted-Average Diluted Shares Outstanding
194.5
197.1
194.1
196.6
Adjustments
Common stock issuable under company's convertible debt (6)
Certain amounts may not sum or recalculate due to rounding.
(2)
Stock-based compensation expense recorded in SG&A for the three and six months ended June 30, 2026, includes approximately $13 million related to the post-combination service period for unvested Amicus stock options.
(3)
These amounts represent costs resulting from the Amicus acquisition that closed on April 27, 2026. Acquisition-related costs were included in SG&A and consisted of severance, transaction and integration costs. Amortization of acquired inventory step-up was included in COS.
(4)
These amounts were included in SG&A and represent charges for severance in connection with the company's plan to simplify its organizational design and strategic initiatives in the first and second quarters of 2026.
(5)
Represents impairment loss on non-marketable equity securities recorded in Other income, net, in the first quarter of 2025.
(6)
Common stock issuable under the company's convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three and six months ended June 30, 2026 as they were anti-dilutive.
Amicus Therapeutics, Inc. (1)
Reconciliation of Non-GAAP Financial Measures
(in thousands)
(Unaudited)
Twelve Months Ended
December 31, 2025
Total operating expenses - as reported GAAP
$ 528,492
Research and development:
Share-based compensation
12,156
Selling, general and administrative:
Share-based compensation
75,254
Loss on impairment of assets
1,702
Depreciation and amortization
7,460
Total operating expense adjustments to reported GAAP
96,572
Total operating expenses - as adjusted
$ 431,920
(1)
The above historical reconciliation is reproduced from Amicus' earnings release furnished as Exhibit 99.1 to its Current Report on Form 8-K dated February 20, 2026 and reflects Amicus' historical definitions of the applicable non-GAAP measures.
Amundi trimmed its position in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 4.6% during the first quarter, according to its most recent Form 13F filing with the Securities & Exchange Commission. The institutional investor owned 188,536 shares of the biotechnology company’s stock after selling 9,047 shares during the period. Amundi owned approximately 0.10% of BioMarin Pharmaceutical worth $10,650,000 as of its most recent SEC filing.
Several other hedge funds and other institutional investors have also modified their holdings of BMRN. Activest Wealth Management purchased a new stake in shares of BioMarin Pharmaceutical during the fourth quarter worth $26,000. CIBC Private Wealth Group LLC raised its holdings in shares of BioMarin Pharmaceutical by 38.2% in the 4th quarter. CIBC Private Wealth Group LLC now owns 648 shares of the biotechnology company’s stock valued at $39,000 after purchasing an additional 179 shares in the last quarter. Caitong International Asset Management Co. Ltd purchased a new stake in shares of BioMarin Pharmaceutical during the 3rd quarter worth $40,000. V Square Quantitative Management LLC purchased a new stake in shares of BioMarin Pharmaceutical during the 4th quarter worth $45,000. Finally, Parallel Advisors LLC grew its stake in shares of BioMarin Pharmaceutical by 62.2% during the third quarter. Parallel Advisors LLC now owns 769 shares of the biotechnology company’s stock worth $42,000 after purchasing an additional 295 shares in the last quarter. 98.71% of the stock is currently owned by hedge funds and other institutional investors.
BioMarin Pharmaceutical Price Performance BioMarin Pharmaceutical stock opened at $60.72 on Wednesday. The company has a debt-to-equity ratio of 0.23, a current ratio of 5.81 and a quick ratio of 4.20. The business has a 50 day simple moving average of $57.74 and a 200-day simple moving average of $56.93. BioMarin Pharmaceutical Inc. has a 12 month low of $49.26 and a 12 month high of $66.28. The stock has a market capitalization of $11.74 billion, a PE ratio of 44.32, a price-to-earnings-growth ratio of 0.46 and a beta of 0.24.
Insider Activity at BioMarin Pharmaceutical In related news, EVP Gregory R. Friberg sold 3,281 shares of BioMarin Pharmaceutical stock in a transaction that occurred on Thursday, May 7th. The stock was sold at an average price of $53.85, for a total value of $176,681.85. Following the sale, the executive vice president directly owned 51,818 shares in the company, valued at $2,790,399.30. The trade was a 5.95% decrease in their ownership of the stock. The transaction was disclosed in a filing with the Securities & Exchange Commission, which is available through this link. 0.68% of the stock is owned by corporate insiders.
Analyst Upgrades and Downgrades A number of research firms have weighed in on BMRN. Bank of America cut their price target on BioMarin Pharmaceutical from $85.00 to $80.00 and set a “buy” rating on the stock in a research note on Tuesday, May 19th. Wall Street Zen lowered BioMarin Pharmaceutical from a “buy” rating to a “hold” rating in a research report on Saturday, June 6th. Weiss Ratings upgraded BioMarin Pharmaceutical from a “sell (d+)” rating to a “hold (c-)” rating in a research note on Friday, June 26th. Sanford C. Bernstein dropped their price target on shares of BioMarin Pharmaceutical from $94.00 to $82.00 and set an “outperform” rating on the stock in a research note on Tuesday, May 5th. Finally, UBS Group restated a “neutral” rating on shares of BioMarin Pharmaceutical in a research report on Tuesday, July 14th. One analyst has rated the stock with a Strong Buy rating, fifteen have assigned a Buy rating and eight have assigned a Hold rating to the company. According to data from MarketBeat, the stock presently has a consensus rating of “Moderate Buy” and an average price target of $87.09.
View Our Latest Analysis on BMRN
About BioMarin Pharmaceutical (Free Report)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
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, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, August 6, 2026, at 4:30 p.m. ET to discuss second quarter 2026 financial results and provide a general business update.
Dial-in Number
U.S. / Canada Dial-in Number: 800-715-9871
International Dial-in Number: 646-307-1963
Conference Call ID: 3551298
U.S. / Canada Replay Dial-in Number: 800-770-2030
International Replay Dial-in Number: 609-800-9909
Playback ID: 3551298
Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, https://investors.biomarin.com/. A replay of the call will be archived on the site for one week following the call.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Wall Street expects a year-over-year decline in earnings on higher revenues when BioMarin Pharmaceutical (BMRN - Free Report) reports results for the quarter ended June 2026. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.
The stock might move higher if these key numbers top expectations in the upcoming earnings report. On the other hand, if they miss, the stock may move lower.
While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise.
Zacks Consensus EstimateThis rare disease biopharmaceutical is expected to post quarterly earnings of $0.96 per share in its upcoming report, which represents a year-over-year change of -33.3%.
Revenues are expected to be $922.33 million, up 11.7% from the year-ago quarter.
Estimate Revisions TrendThe consensus EPS estimate for the quarter has remained unchanged over the last 30 days. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period.
Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggregate change.
Price, Consensus and EPS Surprise
Earnings WhisperEstimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. This insight is at the core of our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction).
The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier.
Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only.
A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP.
Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell).
How Have the Numbers Shaped Up for BioMarin?For BioMarin, the Most Accurate Estimate is lower than the Zacks Consensus Estimate, suggesting that analysts have recently become bearish on the company's earnings prospects. This has resulted in an Earnings ESP of -8.61%.
On the other hand, the stock currently carries a Zacks Rank of #3.
So, this combination makes it difficult to conclusively predict that BioMarin will beat the consensus EPS estimate.
Does Earnings Surprise History Hold Any Clue?While calculating estimates for a company's future earnings, analysts often consider to what extent it has been able to match past consensus estimates. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number.
For the last reported quarter, it was expected that BioMarin would post earnings of $0.94 per share when it actually produced earnings of $0.76, delivering a surprise of -19.15%.
Over the last four quarters, the company has beaten consensus EPS estimates three times.
Bottom LineAn earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss.
That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported.
BioMarin doesn't appear a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release.
An Industry Player's Expected ResultsPTC Therapeutics (PTCT - Free Report) , another stock in the Zacks Medical - Biomedical and Genetics industry, is expected to report loss per share of $0.17 for the quarter ended June 2026. This estimate points to a year-over-year change of +79.5%. Revenues for the quarter are expected to be $294.92 million, up 64.9% from the year-ago quarter.
Over the last 30 days, the consensus EPS estimate for PTC Therapeutics has been revised 66.7% down to the current level. Nevertheless, the company now has an Earnings ESP of 0.00%, reflecting an equal Most Accurate Estimate.
This Earnings ESP, combined with its Zacks Rank #3 (Hold), makes it difficult to conclusively predict that PTC Therapeutics will beat the consensus EPS estimate. Over the last four quarters, the company surpassed consensus EPS estimates three times.
Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar.
ReNU syndrome was discovered in 2024 and there are no medicines approved for the condition
Collaboration combines BioMarin's leadership in genetic medicines with n-Lorem's pioneering antisense expertise
Investigational antisense oligonucleotide (ASO) aims to address the underlying genetic cause of this serious neurodevelopmental condition
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) and n-Lorem Foundation, a nonprofit organization, today announced a strategic collaboration and global exclusive license agreement to develop a first-in-disease, antisense oligonucleotide (ASO) medicine for people living with ReNU syndrome, a serious and rare neurodevelopmental condition caused by variants in the RNU4-2 gene.
Under the agreement, BioMarin and n-Lorem will collaborate to advance an investigational ASO candidate targeting the RNU4-2 (n.64_65insT) variant, which is estimated to account for approximately 75% of ReNU syndrome cases. ReNU syndrome was first discovered in 2024 by an international team of geneticists led by Dr. Nicola Whiffin at the University of Oxford's Big Data Institute and Dr. Ernest Turro at the Mt. Sinai Icahn School of Medicine. There are currently no approved medicines that address the underlying cause of disease.
Both BioMarin and n-Lorem will conduct preclinical studies and collaborate to select the lead candidate to move forward in clinical studies.
"ReNU syndrome was identified as a distinct genetic condition in 2024, thanks in large part to the pioneering efforts of families, advocates and researchers who helped raise awareness and accelerate understanding of this condition," said Kevin Eggan, Ph.D., Chief Scientific Officer at BioMarin. "For many families, a ReNU diagnosis can finally provide answers, but currently there are no approved medicines that address the underlying cause of the disease. By combining BioMarin's expertise in genetic medicines with n-Lorem's pioneering antisense capabilities, we aim to bring the first treatment option for people living with ReNU syndrome."
The n-Lorem Foundation typically focuses on conditions with a very small number of individuals (approximately 30 people or less) worldwide. When a program has the potential to reach a broader population, the foundation seeks a partner such as BioMarin to support development. In the case of ReNU syndrome, the foundation began its program and accepted a number of patients with RNU4-2 to initiate individualized clinical trials in the coming months. Through this new collaboration, BioMarin will lead the development of the investigational medicine for the wider ReNU syndrome community.
"We are proud to partner with BioMarin, a company that shares our urgency and has the scientific, clinical, and commercial expertise to bring this innovative new medicine to better help people living with ReNU Syndrome globally," said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO of n-Lorem. "Our commitment is to develop ASO medicines and, when we recognize the opportunity to support even more individuals, identify a partner that can advance our medicines to be commercially approved."
ReNU syndrome is a rare genetic neurodevelopmental condition associated with cognitive, language and adaptive behavioral impairments. ReNU syndrome is projected to be one of the leading monogenetic causes for developmental delay and impairment, with an expected global population of approximately 100,000.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
About n-Lorem
n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem received over 475 applications for treatment with more than 275 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. For more information, please visit www.nlorem.org.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: plans and expectations regarding the strategic collaboration and global exclusive license agreement between BioMarin and n-Lorem Foundation to develop a first-in-disease, antisense oligonucleotide (ASO) medicine for people living with ReNU syndrome; prospects and timing of actions relating to preclinical and clinical studies and approvals; and BioMarin's estimates regarding global population with ReNU syndrome as well as the prevalence of the RNU4-2 variant. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of planned preclinical and clinical studies; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
BioMarin Contacts:
Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media
Andrew Villani
BioMarin Pharmaceutical Inc.
(628) 269-7393
Bank of New York Mellon Corp decreased its position in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 35.5% in the first quarter, according to the company in its most recent 13F filing with the Securities and Exchange Commission (SEC). The fund owned 1,144,892 shares of the biotechnology company’s stock after selling 631,362 shares during the period. Bank of New York Mellon Corp owned 0.59% of BioMarin Pharmaceutical worth $64,675,000 at the end of the most recent quarter.
Other hedge funds also recently bought and sold shares of the company. Activest Wealth Management acquired a new position in shares of BioMarin Pharmaceutical during the 4th quarter worth about $26,000. CIBC Private Wealth Group LLC increased its position in BioMarin Pharmaceutical by 38.2% in the 4th quarter. CIBC Private Wealth Group LLC now owns 648 shares of the biotechnology company’s stock worth $39,000 after buying an additional 179 shares during the period. Caitong International Asset Management Co. Ltd bought a new position in BioMarin Pharmaceutical in the 3rd quarter worth approximately $40,000. Parallel Advisors LLC lifted its holdings in shares of BioMarin Pharmaceutical by 62.2% during the third quarter. Parallel Advisors LLC now owns 769 shares of the biotechnology company’s stock worth $42,000 after buying an additional 295 shares during the last quarter. Finally, V Square Quantitative Management LLC acquired a new position in shares of BioMarin Pharmaceutical during the fourth quarter worth approximately $45,000. 98.71% of the stock is currently owned by institutional investors.
BioMarin Pharmaceutical Stock Performance NASDAQ BMRN opened at $58.40 on Thursday. The firm has a market cap of $11.29 billion, a price-to-earnings ratio of 42.63, a PEG ratio of 0.45 and a beta of 0.24. The company has a 50 day moving average of $56.27 and a 200-day moving average of $56.82. The company has a current ratio of 5.81, a quick ratio of 4.20 and a debt-to-equity ratio of 0.23. BioMarin Pharmaceutical Inc. has a 1-year low of $49.26 and a 1-year high of $66.28.
Insiders Place Their Bets In other BioMarin Pharmaceutical news, EVP Gregory R. Friberg sold 3,281 shares of BioMarin Pharmaceutical stock in a transaction on Thursday, May 7th. The stock was sold at an average price of $53.85, for a total value of $176,681.85. Following the completion of the transaction, the executive vice president owned 51,818 shares in the company, valued at approximately $2,790,399.30. The trade was a 5.95% decrease in their position. The sale was disclosed in a document filed with the SEC, which can be accessed through this hyperlink. 0.68% of the stock is owned by corporate insiders.
Analysts Set New Price Targets Several brokerages have recently issued reports on BMRN. The Goldman Sachs Group began coverage on shares of BioMarin Pharmaceutical in a research note on Monday, May 11th. They issued a “neutral” rating and a $69.00 price objective on the stock. Bank of America cut their target price on shares of BioMarin Pharmaceutical from $85.00 to $80.00 and set a “buy” rating for the company in a research note on Tuesday, May 19th. HC Wainwright reissued a “neutral” rating on shares of BioMarin Pharmaceutical in a report on Tuesday, July 14th. Citigroup raised their price target on shares of BioMarin Pharmaceutical from $75.00 to $76.00 and gave the stock a “buy” rating in a research report on Thursday, July 16th. Finally, Wall Street Zen downgraded shares of BioMarin Pharmaceutical from a “buy” rating to a “hold” rating in a report on Saturday, June 6th. One analyst has rated the stock with a Strong Buy rating, fifteen have issued a Buy rating and eight have assigned a Hold rating to the stock. Based on data from MarketBeat, the company presently has a consensus rating of “Moderate Buy” and an average target price of $88.26.
Read Our Latest Stock Analysis on BMRN
BioMarin Pharmaceutical Profile (Free Report)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
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D.A. Davidson & CO. boosted its holdings in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 467.1% during the first quarter, according to the company in its most recent filing with the Securities and Exchange Commission (SEC). The firm owned 44,063 shares of the biotechnology company’s stock after purchasing an additional 36,293 shares during the period. D.A. Davidson & CO.’s holdings in BioMarin Pharmaceutical were worth $2,489,000 at the end of the most recent reporting period.
A number of other hedge funds and other institutional investors have also bought and sold shares of the business. Assenagon Asset Management S.A. boosted its position in shares of BioMarin Pharmaceutical by 312.4% in the 4th quarter. Assenagon Asset Management S.A. now owns 1,419,067 shares of the biotechnology company’s stock worth $84,335,000 after purchasing an additional 1,074,929 shares in the last quarter. Perpetual Ltd increased its position in BioMarin Pharmaceutical by 425.7% during the 4th quarter. Perpetual Ltd now owns 25,621 shares of the biotechnology company’s stock valued at $1,523,000 after buying an additional 20,747 shares in the last quarter. Paragon Capital Management Inc. purchased a new position in BioMarin Pharmaceutical during the fourth quarter valued at $1,191,000. Swedbank AB raised its stake in BioMarin Pharmaceutical by 70.6% during the fourth quarter. Swedbank AB now owns 796,737 shares of the biotechnology company’s stock valued at $47,350,000 after buying an additional 329,794 shares during the last quarter. Finally, LSV Asset Management boosted its holdings in BioMarin Pharmaceutical by 4.3% in the fourth quarter. LSV Asset Management now owns 882,200 shares of the biotechnology company’s stock worth $52,429,000 after acquiring an additional 36,700 shares in the last quarter. Institutional investors own 98.71% of the company’s stock.
Wall Street Analyst Weigh In BMRN has been the subject of several analyst reports. HC Wainwright restated a “neutral” rating on shares of BioMarin Pharmaceutical in a report on Tuesday, July 14th. Royal Bank Of Canada reduced their price objective on shares of BioMarin Pharmaceutical from $66.00 to $62.00 and set a “sector perform” rating for the company in a research note on Tuesday, July 7th. Bank of America decreased their target price on shares of BioMarin Pharmaceutical from $85.00 to $80.00 and set a “buy” rating for the company in a report on Tuesday, May 19th. Sanford C. Bernstein cut their price target on shares of BioMarin Pharmaceutical from $94.00 to $82.00 and set an “outperform” rating on the stock in a report on Tuesday, May 5th. Finally, Morgan Stanley decreased their price objective on shares of BioMarin Pharmaceutical from $120.00 to $119.00 and set an “overweight” rating for the company in a report on Tuesday, May 5th. One research analyst has rated the stock with a Strong Buy rating, fifteen have given a Buy rating and eight have assigned a Hold rating to the company. According to MarketBeat, the company presently has an average rating of “Moderate Buy” and a consensus price target of $88.26.
Check Out Our Latest Stock Analysis on BioMarin Pharmaceutical
BioMarin Pharmaceutical Price Performance Shares of NASDAQ:BMRN opened at $59.11 on Tuesday. BioMarin Pharmaceutical Inc. has a fifty-two week low of $49.26 and a fifty-two week high of $66.28. The business has a 50-day simple moving average of $56.05 and a two-hundred day simple moving average of $56.83. The company has a debt-to-equity ratio of 0.23, a quick ratio of 4.20 and a current ratio of 5.81. The firm has a market cap of $11.42 billion, a price-to-earnings ratio of 43.15, a price-to-earnings-growth ratio of 0.45 and a beta of 0.24.
Insider Buying and Selling at BioMarin Pharmaceutical In related news, EVP Gregory R. Friberg sold 3,281 shares of the stock in a transaction that occurred on Thursday, May 7th. The shares were sold at an average price of $53.85, for a total transaction of $176,681.85. Following the transaction, the executive vice president owned 51,818 shares in the company, valued at $2,790,399.30. This represents a 5.95% decrease in their ownership of the stock. The sale was disclosed in a document filed with the Securities & Exchange Commission, which is available at the SEC website. 0.68% of the stock is owned by insiders.
About BioMarin Pharmaceutical (Free Report)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
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Key Takeaways BMRN's sNDA seeking full Voxzogo approval in achondroplasia was accepted for FDA review.BMRN received accelerated FDA approval for Voxzogo in 2021 and now seeks traditional approval.FDA decision is due on Feb. 28, 2027; Voxzogo may become the first achondroplasia drug to get traditional nod. BioMarin Pharmaceutical (BMRN - Free Report) announced that the FDA has accepted the supplemental new drug application (sNDA) seeking full approval for Voxzogo (vosoritide) in children with achondroplasia, the most common form of dwarfism.
With the FDA accepting the sNDA for review, a decision from the regulatory body is expected on Feb. 28, 2027.
Voxzogo received an accelerated approval from the FDA in 2021 to treat children of all ages with achondroplasia. The drug is approved for similar indications in Europe, Japan and Australia.
The latest sNDA, which seeks to convert Voxzogo’s accelerated approval into full/traditional approval, is based on long-term safety and efficacy data from three ongoing studies, comprising adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span, evaluated over long-term follow-up.
Per management, if approved, Voxzogo would become the first therapy for achondroplasia to convert from accelerated to traditional approval based on comprehensive clinical data, including adult height outcomes and other measures assessed over an extended follow-up period.
BMRN’s Stock PerformanceYear to date, shares of BioMarin have lost 0.4% against the industry’s increase of 4.1%.
Image Source: Zacks Investment Research
BMRN Banks on Voxzogo Amid Growing RivalrySince its launch, Voxzogo has seen rapid uptake, driven by strong prescription demand. BioMarin expects to generate $0.98-$1.03 billion from Voxzogo sales in 2026.
BioMarin is also evaluating Voxzogo in the phase III CANOPY-HCH-3 study for a potential second indication, hypochondroplasia, which is generally considered to be a milder form of achondroplasia. In May 2026, the company reported positive data from this study, which achieved its primary and key secondary endpoints. Based on these results, BioMarin expects to submit a regulatory filing with the FDA in the third quarter of 2026.
If approved, the label expansion could broaden the drug’s addressable market opportunity.
However, the achondroplasia treatment market is becoming increasingly competitive.
In February 2026, the FDA approved Ascendis Pharma’s (ASND - Free Report) Yuviwel for achondroplasia, marking the first direct competitor to Voxzogo. Before the approval of ASND’s Yuviwel, Voxzogo was the only FDA-approved therapy for the condition.
Meanwhile, BridgeBio Pharma (BBIO - Free Report) is preparing to submit a regulatory filing to the FDA in the third quarter of 2026 for its investigational achondroplasia candidate, infigratinib. If approved, BBIO expects a potential launch for infigratinib in early to mid-2027.
Amid increasing competition, the successful development and potential label expansion of Voxzogo into additional indications could meaningfully strengthen BioMarin's long-term growth prospects.
BMRN’s Zacks RankBioMarin currently carries a Zacks Rank #4 (Sell).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Application based on long-term safety and efficacy data from three ongoing studies, including adult height and additional clinical outcomes beyond linear growth, including body proportionality and arm span evaluated over long-term follow-up
FDA PDUFA target action date of Feb. 28, 2027
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for full approval in children with achondroplasia. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Feb. 28, 2027.
"This submission for VOXZOGO is supported by the largest body of evidence for any medicine in achondroplasia, reflecting BioMarin's long-standing commitment to advancing the science of skeletal growth. The clinical data demonstrate meaningful improvements across multiple skeletal growth-related measures beyond annualized growth velocity in children with this condition," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "If approved, VOXZOGO would be the first therapy for achondroplasia to convert from accelerated approval to traditional approval based on a comprehensive clinical data package, including adult height outcomes and other clinical measures evaluated over extended follow-up."
The sNDA submission was supported by substantial long-term safety and efficacy data from three ongoing studies (111-205, 111-208 and 111-302), including clinically meaningful results in growth and improvements across key skeletal growth-related measures, including proportionality and arm span. The full package submitted to the FDA included the longest efficacy and safety data of any medicine studied in achondroplasia.
VOXZOGO received FDA accelerated approval in 2021, a pathway enabling faster patient access based on measures reasonably likely to predict clinical benefit. This sNDA is intended to fulfill the postmarketing requirement to confirm that benefit and convert to full approval, supported by long-term data from three ongoing studies demonstrating clinically meaningful improvements in growth and skeletal health outcomes in children with achondroplasia.
About Achondroplasia
Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.
More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.
For more information about our clinical trials in achondroplasia, hypochondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.
About VOXZOGO (vosoritide)
In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.
VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.
Patient Support Accessing VOXZOGO
BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S. doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care.
To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at [email protected].
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO?
VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO?
The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken?
VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO?
Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see additional safety information in the full Prescribing Information and Patient Information.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin's expectations regarding the submission of its supplemental New Drug Application (sNDA) for VOXZOGO (vosoritide) for full approval in children with achondroplasia, including expectations regarding the Prescription Drug User Fee Act (PDUFA) target action date; the safety profile and potential benefits of VOXZOGO for children with achondroplasia, including benefits beyond height; and the development of BioMarin's VOXZOGO program generally and the continued clinical development of VOXZOGO, including in achondroplasia, hypochondroplasia and other skeletal conditions. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, BioMarin RareConnections® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
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Stock to Watch: BioMarin Pharmaceutical (BMRN - Free Report) San Rafael, CA-based BioMarin Pharmaceutical Inc. focuses on the development and commercialization of treatments for life-threatening severe medical conditions, mainly for children.
BMRN is a #3 (Hold) on the Zacks Rank, with a VGM Score of B.
It also boasts a Value Style Score of B thanks to attractive valuation metrics like a forward P/E ratio of 11.57; value investors should take notice.
Three analysts revised their earnings estimate higher in the last 60 days for fiscal 2026, while the Zacks Consensus Estimate has increased $0.34 to $4.95 per share. BMRN also boasts an average earnings surprise of +71.2%.
With a solid Zacks Rank and top-tier Value and VGM Style Scores, BMRN should be on investors' short list.
Data from an investigator-led study showed treatment with VOXZOGO resulted in sustained improvements in growth over three years in children with hypochondroplasia
New Phase 1 results also presented for investigational BMN 333 support potential weekly dosing in children with achondroplasia
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced new data from studies of VOXZOGO® (vosoritide) in children with hypochondroplasia and the investigational medicine BMN 333 in achondroplasia were presented at ENDO 2026, the Endocrine Society Annual Meeting, in Chicago.
New data from a Phase 2, investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and investigators at Children's National Hospital, demonstrated sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS), with a favorable safety profile. Mean height SDS improved by 0.72 SD over three years of treatment, while mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through years two and three.
BioMarin recently announced positive topline results from CANOPY-HCH-3, its registration-enabling Phase 3 pivotal study evaluating VOXZOGO in children with hypochondroplasia. These results will be included in the supplemental New Drug Application submission to the U.S. Food and Drug Administration planned for the third quarter of 2026.
"Building on the excellent Phase 3 results we recently announced, these new longer-term data further reinforce the potential of VOXZOGO to meaningfully improve growth outcomes for children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Importantly, we continue to observe sustained growth improvements over time and a favorable safety profile, adding to the growing body of evidence supporting VOXZOGO for this potential new indication."
New BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development
Additional data being presented at ENDO 2026 highlight the potential of investigational BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy adults, BMN 333 demonstrated sustained systemic exposure associated with prolonged pharmacodynamic target engagement, supporting a weekly dosing schedule. The maximum examined dose of BMN 333 (500 μg/kg) increased exposure to free CNP by more than 13 times compared to another long-acting CNP agent, reflecting the potential of BMN 333 to become the new standard of care in achondroplasia. BMN 333 was well tolerated across all dose levels evaluated, with no dose-limiting toxicities or treatment-related serious adverse events.
In April, BioMarin began enrolling patients in the registration-enabling Phase 2/3 study of BMN 333. A data update from the dose-finding segment of this study is expected in 2027.
Below are key presentations for achondroplasia and hypochondroplasia at ENDO, with all times listed in Central Daylight Time:
Vosoritide Treatment in Children With Hypochondroplasia: Three-Year Results From a Phase 2 Extension Trial
Oral Presentation #ORF47-08
Monday, June 15, 3 – 3:15 p.m.
BMN 333 Achieves High Sustained Released Vosoritide Exposure With Favorable Safety: Phase 1 Results That Support Phase 2/3 Trials in Achondroplasia
Poster Presentation #SUN-212
Sunday, June 14, 9 a.m. – 4 p.m.
About Achondroplasia
Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.
More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.
About Hypochondroplasia
Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings. BioMarin estimates that roughly 14,000 children with hypochondroplasia within the company's global footprint may be eligible for treatment with VOXZOGO.
There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia.
For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.
About VOXZOGO
In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.
VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.
The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency.
Patient Support Accessing VOXZOGO
BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S. doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care.
To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at [email protected].
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO?
VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO?
The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken?
VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO?
Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see additional safety information in the full Prescribing Information and Patient Information.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at ENDO 2026, the Endocrine Society Annual Meeting, including oral and poster presentations; BioMarin's plans and expectations for the development of VOXZOGO for children with hypochondroplasia, including safety profile and potential benefits and plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) during the third quarter of 2026; BioMarin's plans and expectations for the development of BMN 333 for children with achondroplasia, including plans to provide a data update from the dose finding segment of the registration-enabling Phase 2/3 study of BMN 333 in 2027; the safety profile and potential benefits of BMN 333, including the potential to become the new standard of care in achondroplasia; and BioMarin's estimate regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products, including hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, BioMarin RareConnections® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
Pre-Market Stock Futures: The futures are trading lower as we get set to start a new trading week, and after last week’s string of record highs, it may be tough to duplicate the stellar performance that Wall Street put on. All of the major indices closed higher, except the Dow Jones Industrials, which finished the day down 0.16% at 49,230. The Nasdaq continued its hot streak, closing Friday at 24,836, up 1.63% for its fourth straight week of gains, and hitting yet another new all-time high. The S&P 500 did the same, closing at 7,165, up 0.80%, and also hitting another new all-time high. The small-cap Russell 2000, which is still the top-performing index in 2026, up over 11% on the year, closed Friday at 2,787, up 0.43%.
Treasury Bonds: Yields were down across the Treasury curve on Friday as buyers finally returned to U.S. sovereign debt. Everything from the case against Chairman Powell and the Fed being dropped, to the new Fed Chairman Kevin Warsh’s push for new inflation guidelines, to another meeting in Pakistan between our government and Iran for peace talks, was cited as a reason for the buying. When the smoke cleared on Friday, the 30-year-long bond closed at a 4.92% yield, while the benchmark 10-year note closed at 4.31%.
Oil and Gas: Prices were mixed across the energy complex on Friday, as news that peace negotiators were heading to Pakistan for renewed talks boosted hopes for a settlement to the war with Iran, only to be tamped down over the weekend. West Texas Intermediate finished the day down 1.54% at $94.37, while Brent Crude closed higher at $105.40, up 0.29%. Both of these moves came after JPMorgan said oil prices still had room to rise. Natural gas closed down 3.86% at $2.51.
Gold: The precious metals finished up a wild rollercoaster week after published data indicated that central governments around the globe are still buying gold at a breathtaking pace. Gold closed Friday trading at $4,707, up 0.34%, while Silver was last seen at $75.74, up 0.57%.
Crypto: The cryptocurrency markets saw a slight pullback on Friday, with the total market cap dipping 1.35% to $2.57 trillion. Bitcoin held near 11-week highs around $78,000 to $78,300, stalling after a recent rally. The market faces pressure from rising oil prices above $100 and the usual thin weekend liquidity, as cryptocurrencies trade 24/7/365, with Ethereum dipping and traders staying cautious. At 8 AM EDT, Bitcoin was trading at $77,840, while Ethereum was quoted at $2, 321.
24/7 Wall St. reviews dozens of analyst research reports daily to identify new investment ideas for both investors and traders. Some of these daily analyst calls cover stocks to buy. Other calls cover stocks to sell or avoid. Remember that no single analyst call should ever be used as a basis to buy or sell a stock.
Here are some of the top Wall Street analyst upgrades, downgrades, and initiations seen on Monday April, 27, 2026.
Upgrades: CrowdStrike Holdings (NASDAQ: CRWD | CRWD Price Prediction) was upgraded to Outperform from Neutral at Mizuho, which raised the target price for the cybersecurity giant to $520 from $490. Fortinet (NASDAQ: FTNT) was upgraded to Buy from Neutral at Arete, with a $104 target price. Nokia (NYSE: NOK) was upgraded to Buy from Hold at Argus, which has a $15 target price for the company. Rollins (NYSE: ROL) was upgraded to Buy from Neutral at Rotchschild & Co. Redburn, which raised the target price for the stock to $66 from $51.90. Snap (NYSE: SNAP) was raised to Buy from Neutral at Rothschild & Co Redburn, which doubled the target price for the stock to $10 from $5. Downgrades: Adobe (NASDAQ: ADBE) was downgraded to Neutral from Outperform at Mizuho, with a $270 target price. Advanced Micro Devices (NASDAQ: AMD) was downgraded to Market Perform from Outperform at Northland, which has a $260 target price for the legacy chip leader. Digital Realty Trust (NYSE: DLR) was downgraded to Hold from Buy at HSBC, which actually bumped the price target for the datacenter giant to $210 from $193. GE Vernova (NYSE: GEV) was downgraded to Neutral from Outperform at BNP Paribas, with an $1,190 target price. Pinterest (NYSE: PINS) was cut to Neutral from Buy at Rothschild & Co Redburn, which nudged the target price for the shares to $23 from $17. Initiations: BioMarin Pharmaceutical (NASDAQ: BMRN) was resumed in coverage at Morgan Stanley, which raised the target price for the stock to $120 from $98. Cohu (NASDAQ: COHU) was initiated with a Buy rating at Jefferies, which has a $55 target price for the shares. DoorDash (NASDAQ: DASH) was initiated with a Buy rating at TD Cowen, with a $225 target price. Riot Platforms (NASDAQ: RIOT) was initiated with a Buy rating at Chardan, with a $27.50 target price. StubHub Holdings (NYSE: STUB) was started with an Equal Weight rating at Morgan Stanley with an $8.25 target price.
Acquisition Adds Galafold® (migalastat) for Fabry Disease and Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) for Pompe Disease to BioMarin's Commercial Portfolio
BioMarin Expects to Provide Updated FY 2026 Guidance During its First Quarter Earnings Call, May 4, 2026
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) said today that it completed the previously announced agreement to acquire Amicus Therapeutics for $14.50 per share in an all-cash transaction for a total equity value of approximately $4.8 billion. The acquisition will strengthen BioMarin's commercial portfolio, adding two new treatments to the company's existing portfolio of medicines that target lysosomal storage diseases: Galafold® (migalastat), the first oral treatment for Fabry disease, and Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat), a two-component therapy for Pompe disease. BioMarin also now has U.S. rights to DMX-200, a potential first-in-class investigational small molecule for the treatment of focal segmental glomerulosclerosis (FSGS), a rare and fatal kidney disease in Phase 3 development.
"The completion of the Amicus acquisition advances BioMarin's strategy to strengthen and diversify our growth profile while furthering our mission to deliver medicines for people living with rare diseases," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "BioMarin's global scale, established commercial infrastructure, and advanced in‑house manufacturing capabilities build on Amicus' legacy and position us to bring Galafold and Pombiliti + Opfolda to more patients around the world."
About Galafold
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body's own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.
U.S. INDICATIONS AND USAGE
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable GLA variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
U.S. IMPORTANT SAFETY INFORMATION
ADVERSE REACTIONS: The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS: There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus. It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition. Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. The safety and effectiveness of Galafold have not been established in pediatric patients. To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that's designed to reduce loss of enzyme activity in the blood.
U.S. INDICATIONS AND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase (GAA) deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).
SAFETY INFORMATION
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See the full U.S. Prescribing Information for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see U.S. full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) and full PRESCRIBING INFORMATION for OPFOLDA (miglustat).
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about, among other things, the business prospects of Amicus Therapeutics (Amicus) and BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the prospective benefits of the acquisition; expectations regarding Amicus' products, Galafold and Pombiliti + Opfolda; expectations regarding Amicus' product candidate, DMX-200, and its ongoing development; BioMarin's capital allocation strategy to leverage its financial strength to diversify its pipeline and add innovative new therapies for patients; BioMarin's plans for external innovation, including BioMarin's ability to execute additional transactions in future quarters; statements about BioMarin's future performance; and other statements that are not historical facts. Actual results could differ materially from those anticipated in these forward-looking statements. Except as required by law, each of BioMarin and Amicus assume no obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise. These statements, which represent each of BioMarin's and Amicus' current expectations or beliefs concerning various future events that are subject to significant risks and uncertainties, may contain words such as "may," "will," "would," "could," "expect," "anticipate," "intend," "plan," "believe," "estimate," "project," "seek," "should," "strategy," "future," "opportunity," "potential" or other similar words and expressions indicating future results.
These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. Forward-looking statements reflect current beliefs and expectations; however, these statements involve inherent risks and uncertainties, including, without limitation, with respect to: the effects of the acquisition on Amicus' or BioMarin's stock price and/or Amicus' or BioMarin's operating results; unknown or inestimable liabilities; the development, launch and commercialization of products and product candidates; the parties' ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period and that BioMarin and Amicus will not be integrated successfully or that such integration may be more difficult, time-consuming or costly than expected; obtaining and maintaining adequate coverage and reimbursement for BioMarin's or Amicus' products; the time-consuming and uncertain regulatory approval process; the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success, including risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients, including with respect to current and planned future clinical trials; global economic, financial, and healthcare system disruptions and the current and potential future negative impacts to BioMarin's or Amicus' business operations and financial results; the sufficiency of BioMarin's or Amicus' cash flows and capital resources; BioMarin's evaluation of the potential impact of the transaction on its financial results and financial guidance; BioMarin's or Amicus' ability to achieve targeted or expected future financial performance and results and the uncertainty of future tax, accounting and other provisions and estimates; the effects of the transaction on relationships with key third parties, including employees, customers, suppliers, other business partners or governmental entities, including the risk that the acquisition adversely affects employee retention; risks that the acquisition disrupts current plans and operations; any legal proceedings related to the acquisition; and other risks and uncertainties affecting BioMarin and Amicus, including those risk factors detailed in BioMarin's and Amicus' filings with the Securities and Exchange Commission (SEC), including, without limitation, the risk factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the fiscal year ended December 31, 2025 and Amicus' Annual Report on Form 10-K for the fiscal year ended December 31, 2025, as such risk factors may be updated by any subsequent reports, as well as the Proxy Statement on Schedule 14A filed by Amicus (as amended and/or supplemented). Stockholders of BioMarin and Amicus are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin and Amicus are under no obligation, and expressly disclaim any obligation, to update (publicly or otherwise) or alter any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events or otherwise.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc. or its affiliates.
Contacts:
Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media
Marni Kottle
BioMarin Pharmaceutical Inc.
(415) 218-7111
Wall Street expects a year-over-year increase in earnings on higher revenues when Exelixis (EXEL - Free Report) reports results for the quarter ended March 2026. While this widely-known consensus outlook is important in gauging the company's earnings picture, a powerful factor that could impact its near-term stock price is how the actual results compare to these estimates.
The earnings report, which is expected to be released on May 5, might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower.
While management's discussion of business conditions on the earnings call will mostly determine the sustainability of the immediate price change and future earnings expectations, it's worth having a handicapping insight into the odds of a positive EPS surprise.
Zacks Consensus EstimateThis drug developer is expected to post quarterly earnings of $0.75 per share in its upcoming report, which represents a year-over-year change of +21%.
Revenues are expected to be $612.57 million, up 10.3% from the year-ago quarter.
Estimate Revisions TrendThe consensus EPS estimate for the quarter has been revised 1.13% lower over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period.
Investors should keep in mind that an aggregate change may not always reflect the direction of estimate revisions by each of the covering analysts.
Price, Consensus and EPS Surprise
Earnings WhisperEstimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core.
The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier.
Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only.
A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP.
Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell).
How Have the Numbers Shaped Up for Exelixis?For Exelixis, the Most Accurate Estimate is higher than the Zacks Consensus Estimate, suggesting that analysts have recently become bullish on the company's earnings prospects. This has resulted in an Earnings ESP of +6.26%.
On the other hand, the stock currently carries a Zacks Rank of #3.
So, this combination indicates that Exelixis will most likely beat the consensus EPS estimate.
Does Earnings Surprise History Hold Any Clue?Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number.
For the last reported quarter, it was expected that Exelixis would post earnings of $0.77 per share when it actually produced earnings of $0.94, delivering a surprise of +22.08%.
Over the last four quarters, the company has beaten consensus EPS estimates four times.
Bottom LineAn earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss.
That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported.
Exelixis appears a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release.
An Industry Player's Expected ResultsAnother stock from the Zacks Medical - Biomedical and Genetics industry, BioMarin Pharmaceutical (BMRN - Free Report) , is soon expected to post earnings of $1 per share for the quarter ended March 2026. This estimate indicates a year-over-year change of -11.5%. Revenues for the quarter are expected to be $764.28 million, up 2.6% from the year-ago quarter.
The consensus EPS estimate for BioMarin has been revised 4.7% lower over the last 30 days to the current level. However, a lower Most Accurate Estimate has resulted in an Earnings ESP of -5.12%.
This Earnings ESP, combined with its Zacks Rank #3 (Hold), makes it difficult to conclusively predict that BioMarin will beat the consensus EPS estimate. The company beat consensus EPS estimates in each of the trailing four quarters.
Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar.
VOXZOGO is the only approved treatment for children with achondroplasia starting at birth, with over 10 years of clinical research demonstrating the long-term benefit on complications associated with achondroplasia
Researchers will present additional data from studies of VOXZOGO for hypochondroplasia, ahead of pivotal topline Phase 3 data expected in the first half of 2026
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new research from studies of VOXZOGO® (vosoritide) in children with achondroplasia demonstrating positive impact on important health indicators, including arm span and bone density. The data will be presented, along with new data from studies of VOXZOGO in hypochondroplasia, at the Pediatric Endocrine Society's 2026 Annual Meeting (PES) in San Francisco.
Long-Term Treatment Leads to Meaningful Improvement in Multiple Health Measures
Data from three ongoing long-term extension clinical trials demonstrated the impact of long-term treatment with VOXZOGO on measures beyond height, including arm span and bone health. Researchers showed that arm span Z-scores improved from baseline in all age groups, and the arm span-to-height ratio also remained stable over time, showing treatment resulted in proportional skeletal growth. Children who initiated treatment with VOXZOGO after age 5 also achieved a mean difference in standing height of 10.60 cm after six years of treatment (p<0.0001) and 13.59 cm after eight years of treatment (p<0.0001), compared with untreated natural history cohorts.
"With VOXZOGO, we now have a depth and duration of evidence that is unmatched in the treatment of achondroplasia — providing meaningful insight not only into growth, but into the broader, sustained impact on a child's health," said Bradley Miller, M.D., Ph.D., pediatric endocrinologist at the University of Minnesota Medical School. "When you can see consistent benefits over time, it gives you greater confidence to intervene early and treat with purpose."
Another study with 119 children who received VOXZOGO measured the impact of long-term treatment on bone mineral content (BMC) and bone mineral density (BMD) assessed by dual X-ray absorptiometry (DXA) every year for up to six years. Results showed that BMC increased over time, while BMD Z‑scores remained consistent year over year, demonstrating that bone health was maintained in children who received long-term VOXZOGO treatment.
"We are committed to understanding and sharing the clear impact of long-term treatment with VOXZOGO on health measures beyond height, including arm span and bone density, that are meaningful to the thousands of children around the world receiving this medicine," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "With more than ten years of clinical research now, we have seen again and again a breadth of data that continue to demonstrate that long-term and early treatment are critical to ensure the greatest benefit in children with achondroplasia."
New Research in Hypochondroplasia; Topline Phase 3 Results Expected in 1H 2026
Two studies focused on VOXZOGO in hypochondroplasia will also be presented, including one from a single-arm Phase 2 study conducted by Children's National Hospital that showed a statistically significant improvement in total body minus head BMD of 0.03 g/cm2 (p<0.0001) and BMC of 54.84 g (p<0.0001) after 12 months in children who received the medicine.
The company plans to share topline results from its registration-enabling Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026. If the clinical results are positive, the company plans to submit data to health authorities in the second half of 2026 to seek approval for this new indication.
Below are key poster presentations for achondroplasia and hypochondroplasia at PES, with all times listed in Pacific Daylight Time:
Design of a Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Children With Hypochondroplasia Aged <3 Years
Poster #12, Poster Session 2
Friday, May 1, 7:15 – 8:15 a.m.
Effect of Long-Term Vosoritide Treatment in Pediatric Participants With Achondroplasia on Bone Mineral Density and Bone Mineral Content Measured with Dual X-Ray Absorptiometry
Poster #16, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.
Real-World Long-Term Effectiveness and Safety Outcomes of Vosoritide in Adolescents With Achondroplasia in Japan
Poster #58, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.
Vosoritide Treatment Improves Linear Growth and Absolute Bone Content and Density in Children With Hypochondroplasia: A 12-Month Prospective Study
Poster #40, Poster Session 3
Friday, May 1, 12:30 – 2 p.m.
Effect of Long-Term Vosoritide Treatment on Growth in Children With Achondroplasia in Open-Label, Multicenter Clinical Trials
Poster #32, Poster Session 5
Saturday, May 2, 12:30 – 2 p.m.
Improving Guideline-Directed Management of Achondroplasia: Results of a Pre-Implementation Study
Poster #12, Poster Session 5
Saturday, May 2, 12:30 – 2 p.m.
About Achondroplasia
Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.
More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.
For more information about our clinical trials in achondroplasia, hypochondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.
About VOXZOGO
In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
VOXZOGO is the only approved medicine to support the growth of children with achondroplasia starting from birth, with international consensus guidelines recommending initiation of VOXZOGO as early as possible. First approved in 2021, VOXZOGO has helped more than 5,000 infants and children in more than 50 countries. Through our ongoing studies, BioMarin continues to evaluate VOXZOGO on key clinical endpoints relevant for achondroplasia patients, such as arm span, tibial bowing (leg bowing), body proportionality, spinal morphology (including spinal stenosis) and quality of life measures.
VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.
Patient Support Accessing VOXZOGO
BioMarin's robust support services have ensured a seamless treatment experience, spearheaded by Clinical Coordinators, who have conducted hundreds of trainings for families with achondroplasia since approval. BioMarin provides resources to support families navigating achondroplasia, including a caregiver mentorship program that connects parents with other caregivers, and a U.S. doctor directory that helps families and healthcare professionals identify clinicians experienced in achondroplasia care.
To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected]. For more information about VOXZOGO, please visit www.voxzogo.com. For additional information regarding this product, please contact BioMarin Medical Information at [email protected].
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO?
VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO?
The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken?
VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO?
Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see additional safety information in the full Prescribing Information and Patient Information.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the data to be presented at the Pediatric Endocrine Society's 2026 Annual Meeting, including the key poster presentations; the development of BioMarin's VOXZOGO program, including plans to share topline results from the Phase 3 pivotal clinical trial of VOXZOGO in children with hypochondroplasia (CANOPY-HCH-3) in the first half of 2026 and to submit data to health authorities in the second half of 2026 if such results are positive; the safety profile and potential benefits of VOXZOGO for children with achondroplasia and hypochondroplasia; and the continued clinical development of VOXZOGO in multiple indications. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, BioMarin RareConnections® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
First Quarter 2026 Total Revenues Increased Year-over-year to $766 million
Increased Full-year 2026 Total Revenues Guidance to between $3.825 billion and $3.925 billion, Representing Accelerated Growth Rate of 20% Y/Y at the Midpoint, and Reflecting the Addition of GALAFOLD® and POMBILITI® + OPFOLDA® to BioMarin's Portfolio
Conference Call and Webcast Scheduled Today at 4:30 p.m. ET
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the first quarter ended March 31, 2026.
"With the acquisition of Amicus Therapeutics complete, the addition of GALAFOLD and POMBILITI + OPFOLDA to our commercial portfolio allows us to reach patients with Fabry and Pompe diseases and meaningfully strengthens and accelerates our near-to-mid-term growth rates," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We expect these high-growth assets to support our strongest financial performance yet in 2026. Next quarter, we look forward to updating you on the longer‑term outlook of the Amicus integration based on our plans to leverage our global scale to expand the potential of these transformative therapies. With a faster-growing commercial portfolio, together with two near-term Phase 3 data readouts and ongoing pipeline progress expected over the coming quarters, we are well-positioned to drive innovation, create shareholder value, and improve outcomes for patients worldwide."
2026 Business and Pipeline Highlights
Innovation
In February, U.S. FDA approved PALYNZIQ® for adolescents 12 years of age and older with phenylketonuria (PKU); EU approval for adolescents 12 years of age and older is expected in 2026. In March, the company presented initial Phase 1/2 data for BMN 351 at the Muscular Dystrophy Association (MDA) Clinical & Scientific Congress demonstrating dose-dependent increases in dystrophin expression at Week 25 biopsy in both the 6 and 9 mg/kg dose cohorts. Clinical biomarkers, including decreases in creatine kinase, suggested improvements in overall muscle health beyond the Week 25 time point, and longer-term outcomes from both NSAA and 6MWT suggested a prevention of functional decline when compared to historical matched controls. The 12 mg/kg dose cohort continues to enroll, with a data update expected by year-end. In April, the first patient was enrolled in the registration-enabling Phase 2/3 study of BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. A data update from this study is expected in 2027. In April, the company submitted its U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO® for achondroplasia. The company expects to be notified of sNDA acceptance by Q3 2026. In May, at the Pediatric Endocrine Society's (PES) annual meeting, BioMarin reported new data demonstrating the benefits of long-term treatment with VOXZOGO, including improvements in arm span, bone health, and quality of life. Data from ongoing long-term extension clinical trials showed that children who initiated VOXZOGO treatment after age 5 achieved mean height gains of +10.60cm after six years and +13.59cm (p<0.0001 for both) after eight years of treatment, as compared to natural history data. In the second quarter, BioMarin expects to share BMN 401 Phase 3 topline data in children ages 1-to-12 year-old with ENPP1 deficiency. Regulatory submissions are anticipated in 2H'26 should the data be supportive, with a potential first-in-disease launch in 2027. In the second quarter, the company expects to share Phase 3 topline data for VOXZOGO for hypochondroplasia. Regulatory submissions are anticipated in 2H'26 should the data be supportive, with a potential first-in-class launch in 2027. Enrollment is progressing in the Phase 2 study of VOXZOGO in children under 3 years old with hypochondroplasia. Growth
Increased full-year 2026 Total Revenues guidance, accelerating anticipated growth rate to 20% Y/Y, as a result of the addition of GALAFOLD for Fabry disease and POMBILITI + OPFOLDA to BioMarin's commercial portfolio. Enzyme Therapies revenue grew 6% Y/Y in the first quarter, driven by revenue growth for VIMIZIM®, NAGLAZYME®, and BRINEURA®. Continued underlying patient demand in Q1 for PALYNZIQ was driven by an increase in enrollments and new starts in the under-18-year age group following label expansion in February. PALYNZIQ revenue is expected to increase over time with continued patient demand and as new adult and adolescent patients titrate up to maintenance dosing. As a result, full-year 2026 PALYNZIQ revenue is expected to increase year-over-year. The number of children being treated with VOXZOGO increased by more than 20% Y/Y in the first quarter. As expected, large VOXZOGO orders in fourth quarter of 2025 resulted in modest Y/Y growth of 3% in the first quarter of 2026. Value Commitment
During the first quarter, the company secured financing of approximately $3.7 billion of non-convertible debt to support the Amicus acquisition, achieving favorable pricing across the capital structure. BioMarin generated operating cash flows totaling $221 million in first quarter 2026. Total cash was approximately $2 billion as of the end of the quarter, and continued increasing operating cash flow is expected to support sustained investment in innovation and future growth. First Quarter 2026 Financial Highlights
Total Revenues for the first quarter of 2026 were $766 million, an increase of $21 million compared to the same period in 2025, primarily driven by timing of large government orders outside the U.S. and increase in patient demand for Enzyme Therapies (ALDURAZYME®, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM) as well as new patients initiating VOXZOGO therapy across all regions. The increase was partially offset by lower ROCTAVIAN® revenue attributed to voluntary withdrawal of the product from the market announced in the first quarter of 2026. GAAP Net Income for the first quarter of 2026 decreased to $106 million compared to $186 million for the same period in 2025. The decrease in GAAP Net Income was primarily attributed to the following: higher Selling, General & Administrative (SG&A) spend primarily due to incremental administrative costs related to ongoing support of corporate initiatives and pre-close costs for Amicus acquisition, and higher sales and marketing spend on VOXZOGO, PALYNZIQ and VIMIZIM; higher Cost of Sales primarily due to a $31 million charge associated with an unsuccessful process qualification campaign to expand NAGLAZYME manufacturing capabilities; higher Research and Development spend to support BMN 401, a late-stage clinical program acquired in the third quarter of 2025; partially offset by revenue growth as mentioned above. Non-GAAP Income for the first quarter of 2026 decreased to $149 million compared to $221 million for the same period in 2025. The decrease in Non-GAAP Income was primarily due to the factors noted above. GAAP Diluted Earnings per Share (EPS) and Non‑GAAP Diluted EPS for the first quarter of 2026 decreased compared to the same period in 2025, primarily reflecting the discrete items and higher operating expenses described above. The $31 million charge in Cost of Sales related to the NAGLAZYME campaign reduced EPS by approximately $0.12 year‑over‑year. In addition, pre‑close integration preparation costs recorded in SG&A and interest expense associated with the Amicus transaction reduced EPS by approximately $0.07. Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)
Three Months Ended
March 31,
2026
2025
% Change
Total Revenues
$766
$745
3 %
Net Product Revenues by Product:
VOXZOGO
$220
$214
3 %
Enzyme Therapies:
VIMIZIM
$210
$188
12 %
NAGLAZYME
130
114
14 %
PALYNZIQ
90
93
(3) %
BRINEURA
47
40
18 %
ALDURAZYME
37
49
(24) %
Total Enzyme Therapies Revenue
$514
$484
6 %
KUVAN®
$24
$25
(4) %
ROCTAVIAN
$3
$11
(73) %
GAAP Net Income
$106
$186
(43) %
Non-GAAP Income (1)
$149
$221
(33) %
GAAP Operating Margin % (2)
16.9 %
30.0 %
Non-GAAP Operating Margin % (1)
24.3 %
35.7 %
GAAP Diluted EPS
$0.54
$0.95
(43) %
Non-GAAP Diluted EPS (1)
$0.76
$1.13
(33) %
(1)
Refer to Non-GAAP Information beginning on page 9 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin percentage and Non-GAAP Diluted EPS along with the related reconciliations to the comparable information reported under U.S. GAAP.
(2)
GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues.
Forward-Looking Non-GAAP Financial Information
BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.
Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)
Updated guidance reflects post-close contributions from Amicus beginning April 27, 2026. As previously communicated, the acquisition of Amicus is expected to be slightly dilutive to full-year 2026 Non‑GAAP Diluted EPS; historical BioMarin Non-GAAP Diluted EPS guidance is unchanged. The Amicus acquisition will be accounted for as a business combination, which will result in intangible amortization impacting GAAP results over future periods and excluded from Non‑GAAP results. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Guidance is subject to change based on various factors including finalization of purchase accounting. The company expects approximately two-thirds of 2026 Non-GAAP Diluted EPS to be recognized in the second half of 2026, primarily due to the anticipated timing of revenue (more than 55% of 2026 Total Revenues is expected in 2H). Non-GAAP Diluted EPS in Q2 is expected to be modestly higher than in Q1. Item
Provided on February 23, 2026
Updated May 4, 2026
Total Revenues
$3,325
to
$3,425
$3,825
to
$3,925
Enzyme Therapies
$2,225
to
$2,275
$2,725
to
$2,775
VOXZOGO
$975
to
$1,025
Unchanged
Other Revenues(1)
$100
to
$125
Unchanged
Non-GAAP Diluted EPS (2)(3)(4)
$4.95
to
$5.15
$4.85
to
$5.05
(1)
Other Revenues includes KUVAN, ROCTAVIAN, and royalties.
(2)
Refer to Non-GAAP Information beginning on page 9 of this press release for definition of Non-GAAP Diluted EPS.
(3)
Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.
(4)
Non-GAAP Diluted EPS guidance assumes a combined company tax rate of 22%, which is subject to change as the company completes its integration activities and purchase accounting.
BioMarin will host a conference call and webcast to discuss first quarter 2026 financial results today, Monday, May 4, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.
U.S./Canada Dial-in Number: 800-715-9871
Replay Dial-in Number: 800-770-2030
International Dial-in Number: 646-307-1963
Replay International Dial-in Number: 609-800-9909
Conference ID: 3424435
Conference ID: 3424435
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Diluted EPS and operating cash flow for, in certain instances, the full-year 2026, second quarter and second half of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Enzyme Therapies portfolio, including PALYNZIQ, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the addition of GALAFOLD and POMBILITI + OPFOLDA to BioMarin's portfolio; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products.
These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to realize the anticipated benefits of any acquisitions; BioMarin's ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF INCOME
Three Months Ended March 31, 2026 and 2025
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
Three Months Ended
March 31,
2026
2025
REVENUES:
Net product revenues
$ 760,078
$ 734,644
Royalty and other revenues
6,130
10,501
Total revenues
766,208
745,145
OPERATING EXPENSES:
Cost of sales
194,999
151,558
Research and development
178,796
158,731
Selling, general and administrative
258,290
206,116
Intangible asset amortization
4,483
4,847
Total operating expenses
636,568
521,252
INCOME FROM OPERATIONS
129,640
223,893
Interest income
22,560
19,013
Interest expense
(14,958)
(2,863)
Other income (expense), net
3,961
(1,954)
INCOME BEFORE INCOME TAXES
141,203
238,089
Provision for income taxes
35,676
52,403
NET INCOME
$ 105,527
$ 185,686
EARNINGS PER SHARE, BASIC
$ 0.55
$ 0.97
EARNINGS PER SHARE, DILUTED
$ 0.54
$ 0.95
Weighted average common shares outstanding, basic
192,497
190,967
Weighted average common shares outstanding, diluted
197,671
196,474
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
March 31, 2026 and 2025
(In thousands of U.S. dollars, except per share amounts)
(Unaudited)
March 31, 2026
December 31, 2025
ASSETS
Current assets:
Cash and cash equivalents
$ 2,222,435
$ 1,311,679
Short-term investments
—
248,930
Accounts receivable, net
903,914
908,214
Inventory
1,273,221
1,298,883
Other current assets
205,500
185,784
Total current assets
4,605,070
3,953,490
Noncurrent assets:
Long-term investments
—
492,242
Property, plant and equipment, net
958,071
952,508
Intangible assets, net
204,662
213,837
Goodwill
196,199
196,199
Deferred tax assets
1,500,598
1,508,697
Restricted cash equivalents
850,000
—
Other assets
276,416
277,049
Total assets
$ 8,591,016
$ 7,594,022
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable and accrued liabilities
$ 793,152
$ 759,031
Total current liabilities
793,152
759,031
Noncurrent liabilities:
Long-term debt, net
1,430,282
597,176
Other long-term liabilities
155,475
150,816
Total liabilities
2,378,909
1,507,023
Stockholders' equity:
Common stock, $0.001 par value: 500,000,000 shares authorized; 193,268,870 and
192,300,101 shares issued and outstanding, respectively
193
192
Additional paid-in capital
5,966,868
5,956,582
Company common stock held by the Nonqualified Deferred Compensation Plan
(10,450)
(10,508)
Accumulated other comprehensive income (loss)
(4,237)
(13,473)
Retained earnings
259,733
154,206
Total stockholders' equity
6,212,107
6,086,999
Total liabilities and stockholders' equity
$ 8,591,016
$ 7,594,022
BIOMARIN PHARMACEUTICAL INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
Three Months Ended March 31, 2026 and 2025
(In thousands of U.S. dollars)
(Unaudited)
Three Months Ended March 31,
2026
2025
CASH FLOWS FROM OPERATING ACTIVITIES:
Net income
$ 105,527
$ 185,686
Adjustments to reconcile net income to net cash provided by operating activities:
Depreciation and amortization
16,411
22,069
Non-cash interest expense
6,086
660
Accretion of discount on investments
(455)
(1,362)
Stock-based compensation
43,458
37,700
Impairment of assets
—
2,967
Deferred income taxes
9,220
28,429
Unrealized foreign exchange losses (gains)
6,710
(10,026)
Other
(5,374)
(1,267)
Changes in operating assets and liabilities:
Accounts receivable, net
(7,159)
(57,590)
Inventory
44,490
(24,335)
Other current assets
(11,551)
(6,327)
Other assets
1,484
(1,624)
Accounts payable and accrued liabilities
3,100
(2,655)
Other long-term liabilities
8,704
2,069
Net cash provided by operating activities
220,651
174,394
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property, plant and equipment
(20,923)
(16,768)
Maturities and sales of investments
767,277
77,804
Purchases of investments
(25,792)
(89,274)
Other
4,966
—
Net cash provided by (used in) investing activities
725,528
(28,238)
CASH FLOWS FROM FINANCING ACTIVITIES:
Taxes paid related to net share settlement of equity awards
(28,180)
(38,779)
Proceeds from issuance of debt
850,000
—
Payments of debt issuance costs
(8,653)
—
Net cash provided by (used in) financing activities
813,167
(38,779)
Effect of exchange rate changes on cash
1,410
(1,416)
NET INCREASE IN CASH, CASH EQUIVALENTS AND RESTRICTED CASH
EQUIVALENTS
1,760,756
105,961
Cash, cash equivalents and restricted cash equivalents:
Beginning of period
$ 1,311,679
$ 942,842
End of period
$ 3,072,435
$ 1,048,803
Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP R&D expenses and Non-GAAP SG&A expenses are defined by the company as GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans or convertible debt in periods when they are dilutive under Non-GAAP.
BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business.
Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.
The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:
Reconciliation of GAAP Reported Information to Non-GAAP Information (1)
(In millions of U.S. dollars, except per share data)
(unaudited)
Three Months Ended
March 31,
2026
2025
GAAP Reported Net Income
$ 106
$ 186
Adjustments
Stock-based compensation expense - COS
4
2
Stock-based compensation expense - R&D
12
12
Stock-based compensation expense - SG&A
28
23
Amortization of intangible assets
4
5
Severance costs (2)
8
—
Loss on investments (3)
—
3
Income tax effect of adjustments
(13)
(10)
Non-GAAP Income
$ 149
$ 221
Three Months Ended
March 31,
2026
2025
R&D
SG&A
R&D
SG&A
GAAP expenses
$ 179
$ 258
$ 159
$ 206
Adjustments
Stock-based compensation expense
(12)
(28)
(12)
(23)
Severance costs (2)
—
(8)
—
—
Non-GAAP expenses
$ 167
$ 222
$ 147
$ 183
Three Months Ended
March 31,
2026
Percent
of
GAAP
Total
Revenue
2025
Percent
of
GAAP
Total
Revenue
GAAP Income from Operations
$ 130
16.9 %
$ 224
30.0 %
Adjustments
Stock-based compensation expense
44
5.7
37
5.0
Amortization of intangible assets
4
0.5
5
0.7
Severance costs (2)
8
1.0
—
—
Non-GAAP Income from Operations
$ 186
24.3 %
$ 266
35.7 %
Three Months Ended
March 31,
2026
2025
GAAP Diluted EPS
$ 0.54
$ 0.95
Adjustments
Stock-based compensation expense
0.22
0.19
Amortization of intangible assets
0.02
0.03
Severance costs (2)
0.04
—
Loss on investments (3)
—
0.02
Income tax effect of adjustments
(0.07)
(0.05)
Non-GAAP Diluted EPS(4)
$ 0.76
$ 1.13
(1)
Certain amounts may not sum or recalculate due to rounding.
(2)
These amounts were included in SG&A and represent charges for severance in connection with the company's plan to simplify its organizational design and strategic initiatives in the first quarter of 2026.
(3)
Represents impairment loss on non-marketable equity securities recorded in Other income (expense), net, in the first quarter of 2025.
(4)
Both GAAP and Non-GAAP Weighted-Average Diluted Shares Outstanding were 197.7 million and 196.5 million shares for the three months ended March 31, 2026 and 2025, respectively.
BioMarin Pharmaceutical (BMRN - Free Report) came out with quarterly earnings of $0.76 per share, missing the Zacks Consensus Estimate of $0.94 per share. This compares to earnings of $1.13 per share a year ago. These figures are adjusted for non-recurring items.
This quarterly report represents an earnings surprise of -18.96%. A quarter ago, it was expected that this rare disease biopharmaceutical would post earnings of $0.25 per share when it actually produced earnings of $0.46, delivering a surprise of +84%.
Over the last four quarters, the company has surpassed consensus EPS estimates three times.
BioMarin, which belongs to the Zacks Medical - Biomedical and Genetics industry, posted revenues of $766.21 million for the quarter ended March 2026, surpassing the Zacks Consensus Estimate by 0.50%. This compares to year-ago revenues of $745.15 million. The company has topped consensus revenue estimates three times over the last four quarters.
The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.
BioMarin shares have lost about 9% since the beginning of the year versus the S&P 500's gain of 5.6%.
What's Next for BioMarin?While BioMarin has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?
There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.
Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.
Ahead of this earnings release, the estimate revisions trend for BioMarin was mixed. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #3 (Hold) for the stock. So, the shares are expected to perform in line with the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.
It will be interesting to see how estimates for the coming quarters and the current fiscal year change in the days ahead. The current consensus EPS estimate is $1.21 on $834.21 million in revenues for the coming quarter and $4.97 on $3.35 billion in revenues for the current fiscal year.
Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Medical - Biomedical and Genetics is currently in the bottom 44% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.
Compugen (CGEN - Free Report) , another stock in the same industry, has yet to report results for the quarter ended March 2026.
This drug developer is expected to post quarterly loss of $0.07 per share in its upcoming report, which represents a year-over-year change of +12.5%. The consensus EPS estimate for the quarter has been revised 3.5% higher over the last 30 days to the current level.
Compugen's revenues are expected to be $2.62 million, up 14.9% from the year-ago quarter.
For the quarter ended March 2026, BioMarin Pharmaceutical (BMRN - Free Report) reported revenue of $766.21 million, up 2.8% over the same period last year. EPS came in at $0.76, compared to $1.13 in the year-ago quarter.
The reported revenue represents a surprise of +0.5% over the Zacks Consensus Estimate of $762.4 million. With the consensus EPS estimate being $0.94, the EPS surprise was -18.96%.
While investors closely watch year-over-year changes in headline numbers -- revenue and earnings -- and how they compare to Wall Street expectations to determine their next course of action, some key metrics always provide a better insight into a company's underlying performance.
As these metrics influence top- and bottom-line performance, comparing them to the year-ago numbers and what analysts estimated helps investors project a stock's price performance more accurately.
Here is how BioMarin performed in the just reported quarter in terms of the metrics most widely monitored and projected by Wall Street analysts:
Revenues- Net Product Revenues- ALDURAZYME: $37 million versus $46.11 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a -24.5% change.Revenues- Net Product Revenues- KUVAN: $24 million versus $15.5 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a -4% change.Revenues- Net Product Revenues- NAGLAZYME: $130 million versus $117.67 million estimated by seven analysts on average. Compared to the year-ago quarter, this number represents a +14% change.Revenues- Net Product Revenues- VIMIZIM: $210 million compared to the $193.58 million average estimate based on seven analysts. The reported number represents a change of +11.7% year over year.Revenues- Royalty and other revenues: $6.13 million compared to the $15.58 million average estimate based on seven analysts. The reported number represents a change of -41.6% year over year.Revenues- Net Product Revenues- PALYNZIQ: $90 million compared to the $111.91 million average estimate based on seven analysts. The reported number represents a change of -3.2% year over year.Revenues- Net Product Revenues- VOXZOGO: $220 million versus the seven-analyst average estimate of $216.32 million. The reported number represents a year-over-year change of +2.8%.Revenues- Net product revenues: $514 million compared to the $746.65 million average estimate based on seven analysts. The reported number represents a change of -30% year over year.Revenues- Net Product Revenues- BRINEURA: $47 million versus the seven-analyst average estimate of $45.9 million. The reported number represents a year-over-year change of +17.5%.View all Key Company Metrics for BioMarin here>>>
Shares of BioMarin have returned -2.6% over the past month versus the Zacks S&P 500 composite's +10% change. The stock currently has a Zacks Rank #3 (Hold), indicating that it could perform in line with the broader market in the near term.
Key Takeaways BioMarin reported Q1 EPS of 76 cents, missing estimates, while revenue rose 3% to $766.2M.BMRN earnings fell 33% due to a $31M charge and higher costs tied to the Amicus acquisition.BioMarin raised its 2026 revenue outlook to $3.83-$3.93B, reflecting contributions from new therapies. BioMarin Pharmaceutical (BMRN - Free Report) reported first-quarter 2026 adjusted earnings per share of 76 cents, missing the Zacks Consensus Estimate of 94 cents. However, earnings declined 33% year over year. This was largely due to a $31 million charge tied to the company’s unsuccessful campaign to extend Naglazyme manufacturing capabilities, as well as higher operating expenses associated with the acquisition of Amicus Therapeutics.
Total revenues in the first quarter were $766.2 million, up 3% year over year. The figure beat the Zacks Consensus Estimate of $762.4 million.
BMRN Stock MovementShares of BioMarin were down in after-hours trading on Monday, likely due to the mixed earnings results.
Year to date, the stock has lost about 7% compared with the industry’s 2% decline.
Image Source: Zacks Investment Research
More on BMRN’s EarningsNet product revenues totaled nearly $760.1 million, up 3.5% year over year on higher revenues from the company’s Enzyme Therapies, as well as Voxzogo.
Royalty and other revenues totaled $6.1 million, down about 42% year over year.
Voxzogo, approved for achondroplasia, generated sales of $220 million, up 3% year over year. Per the company, this modest upside was expected, as it had previously experienced large orders for the drug in the fourth quarter of 2025. Despite this, Voxzogo sales beat the Zacks Consensus Estimate of $216 million.
BioMarin reports consolidated revenues from five products — Aldurazyme, Brineura, Naglazyme, Palynziq and Vimizim — under a single segment, “Enzyme Therapies.” Sales from this franchise increased 6% year over year to $514 million in the reported quarter, driven by higher product sales of Vimizim, Naglazyme and Brineura.
Palynziq injection sales totaled $90 million in the quarter, down 3% year over year, impacted by order timing in the United States. The drug’s sales missed the Zacks Consensus Estimate of $112 million.
Vimizim sales rose 12% year over year to $210 million, which beat the Zacks Consensus Estimate of $194 million.
Naglazyme sales increased 14% year over year to $130 million. Brineura generated sales of $47 million, up 18%.
Product revenues from Aldurazyme totaled $37 million, down 24% year over year.
BioMarin signed a collaboration agreement with Sanofi’s (SNY - Free Report) subsidiary, Genzyme, for Aldurazyme. SNY, through Genzyme, is BMRN’s sole customer for Aldurazyme. The Sanofi subsidiary is responsible for marketing and selling Aldurazyme to third parties.
Other RevenuesThe gene therapy Roctavian generated $3 million in sales compared with $11 million in the year-ago period. This downside is attributable to the company’s decision to voluntarily withdraw the product from the market.
In the phenylketonuria (PKU) franchise, Kuvan revenues declined 4% to $24 million due to generic competition. The drug lost U.S. market exclusivity in late 2020.
BMRN’s 2026 OutlookLast week, BioMarin announced that it completed the acquisition of Amicus Therapeutics for $4.8 billion. Post-acquisition, the company added two marketed therapies — Galafold (for Fabry disease) and Pombiliti-Opfolda (a combination therapy for Pompe disease) — which will form part of the Enzyme Therapies segment.
BMRN now expects to record total revenues in the range of $3.83-$3.93 billion in 2026, up from the previous guidance of $3.33-$3.43 billion. This new guidance, which includes contributions from Amicus’ marketed drugs, suggests growth of 20% at the mid-point of the range. Management expects to generate more than 55% of the overall 2026 revenues in the second half of the year.
While BioMarin reiterated its Voxzogo sales guidance to be in the range of $975 million to $1.03 billion, it now expects enzyme therapies revenues to be between $2.73 billion and $2.78 billion (previously: $2.23-$2.28 billion). Despite the decline in first-quarter sales, BMRN expects Palynziq sales to increase in 2026, primarily boosted by the drug’s recent approval in adolescents with PKU.
Since the company has accounted for the Amicus Therapeutics acquisition as a business combination, it will result in intangible amortization impacting GAAP results over future periods and will be excluded from non-GAAP results. However, both these results will be impacted by interest expense related to the Amicus financing.
BioMarin has revised its adjusted earnings per share (EPS) to be in the range of $4.85-$5.05 for the year, down from the previous guidance of $4.95-$5.15. While the company expects about two-thirds of this figure to be recognized in the second half of the year, it projects the adjusted EPS in the second quarter to be modestly higher than in the first quarter.
BMRN’s Recent Pipeline UpdatesBioMarin continues to advance its CANOPY clinical program, which evaluates Voxzogo in a phase III study for a potential second indication — hypochondroplasia, a condition characterized by impaired bone growth. Data from this study is expected in the second quarter of 2026, while regulatory submissions are expected thereafter in the second half of 2026 (provided the data is supportive).
As part of the CANOPY program, BMRN is also evaluating Voxzogo in separate phase II studies for two other short-stature pathway conditions — idiopathic short stature and Noonan Syndrome.
The company also expects to report data in the second quarter of 2026 from another phase III study evaluating BMN 401 for the treatment of a rare genetic disorder called ENPP1 deficiency in children. If this data is supportive, BioMarin intends to start regulatory submissions for the therapy in the second half of the year.
Last month, BMRN enrolled the first patient in a registration-enabling phase II/III study evaluating BMN 333 — a long-acting formulation of CNP — as a potential treatment for multiple growth-related conditions that offers the option for less frequent dosing. An update on this candidate is expected next year.
BMRN’s Zacks RankBioMarin currently carries a Zacks Rank #3 (Hold).
Stocks to ConsiderSome better-ranked stocks include Castle Biosciences (CSTL - Free Report) and Catalyst Pharmaceuticals (CPRX - Free Report) . While CSTL sports a Zacks Rank #1 (Strong Buy) at present, CPRX carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, estimates for Castle Biosciences’ 2026 loss per share have narrowed from $1.42 to $1.40. Over the same period, loss per share estimates for 2027 have narrowed from 79 cents to 78 cents. CSTL shares have lost 34% year to date.
Castle Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 34.69%.
Over the past 30 days, estimates for Catalyst Pharmaceuticals’ 2026 EPS have risen from $2.78 to $2.79. Over the same period, EPS estimates for 2027 have increased from $3.25 to $3.28. CPRX shares have gained 24% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
BioMarin Pharmaceutical remains a "Strong Buy," driven by pipeline progress, strategic acquisitions, and robust revenue guidance. BMRN's acquisition of Amicus Therapeutics adds GALAFOLD and POMBILITI + OPFOLDA, boosting 2026 revenue guidance to $3.825–$3.925 billion and targeting 20% YoY growth. VOXZOGO expansion into hypochondroplasia and ongoing clinical trials represent major catalysts, with topline Phase 3 data expected in Q2 2026.
Pre-Market Stock Futures: Futures are trading lower as we get set to start the new trading week, as reports indicate that President Trump declined Iran’s counteroffer for peace. This comes after a remarkable Friday, when stocks roared to record highs, driven primarily by a stronger-than-expected April jobs report that eased economic concerns and by a rally in technology and chip stocks. The S&P 500 rose 0.8% to close the session at 7,398 and notched its longest winning streak since 2024, while the Nasdaq Composite climbed 1.7% and finished the day at 26,247, both marking their sixth consecutive week of gains. The legacy Dow Jones Industrial Average eked out a small gain to close at 49,609, while the small-cap-heavy Russell 2000 was last seen at 2,861, up 0.71%. Earnings for the first quarter, which have been outstanding, are all but over, and Wall Street’s focus will remain on oil prices, the war in Iran, and any indications that inflation is edging higher.
Treasury Bonds: After briefly touching the 5% and higher level for long-dated Treasury bonds early last week, the buyers were once again the story on Friday as yields acros the curve were lower. The solid job numbers were cited as the reason buyers were looking past the inflationary implications of higher oil prices and increased Treasury borrowing. The 30-year long bond was last seen at 4.94% while the benchmark 10-year note finished the day at 4.36%.
Oil and Gas: A modest uptick in oil prices was among Friday’s few negatives. Analysts pointed to fresh military hostilities in the Strait of Hormuz, where U.S. and Iranian forces traded fire, putting the already fragile ceasefire at risk and stoking fears that supply flows through the vital Middle Eastern shipping lane could be disrupted for longer than anticipated. Brent Crude was last seen at $101.30, up 1.23%, while West Texas Intermediate finished the session at $95.42, up 0.64%. Natural gas closed Friday at $2.75, down 0.43%.
Gold: Gold moved in lockstep with stocks and bonds on Friday, capping one of its strongest weeks in recent memory. The precious metal settled at $4,713, its highest close since April 22, and logged a weekly gain of more than 2%, lifted by growing optimism that a U.S.-Iran peace deal could take shape and relieve the inflation pressure that has kept rate-cut hopes at bay. That backdrop is worth keeping in mind: gold had shed more than 10% since the war broke out in late February, dragged lower by surging oil prices that fanned inflation fears and pushed back expectations for easier monetary policy. At the margin, Friday’s bounce looks more like a peace-driven relief rally than a classic flight to safety. Silver also closed higher, and was last seen Friday at $80.22, up 2.47%.
Crypto: Crypto markets pulled back Friday as momentum stalled following a recent surge. Bitcoin slipped to around $80,000, down 0.8% on the day, a modest retreat, but enough to signal cooling sentiment across the board with major assets like Ethereum, Solana, and XRP also trailing off their weekly highs. At 8 AM EDT, Bitcoin was trading at $81,131, while Ethereum was quoted at $2,334.
24/7 Wall St. reviews dozens of analyst research reports daily to identify new investment ideas for both investors and traders. Some of these daily analyst calls cover stocks to buy. Other calls cover stocks to sell or avoid. Remember that no single analyst call should ever be used as a basis to buy or sell a stock.
Here are some of the top Wall Street analyst upgrades, downgrades, and initiations seen on Monday, May 11, 2026.
Upgrades: NatWest Group (NYSE: NWG | NWG Price Prediction) was upgraded to Outperform from Neutral at BNP Paribas, with an $18.20 target price. News Corporation (NASDAQ: NWSA) was raised to Outperform from Market Perform at Macquarie, with a $29.40 target price. Pitney Bowes (NYSE: PBI) was upgraded to Neutral from Underperform at Bank of America, which raised the price target to $16.50 from $9.50. Primoris Services (NYSE: PRIM) was raised to Outperform from Neutral at Mizuho, which lowered the target price for the stock to $135 from $175. Walt Disney (NYSE: DIS) was upgraded to Buy from Hold at Phillip Securities, with a $139 target price. Downgrades: Dell Technologies (NYSE: DELL) was downgraded to Neutral from Buy at UBS, with a $243 target price for the stock, up from $167. Healthpeak Properties (NYSE: DOC) was downgraded to Inline from Outperform at Evercore ISI, which nudged the price target for the stock to $21 from $20. HubSpot (NYSE: HUBS) was downgraded to Neutral from Outperform at Macquarie, which slashed the target price for the stock to $190 from $350. Trade Desk (NASDAQ: TTD) was cut to Reduce from Hold at HSBC with a $20 target price. Wendy’s (NYSE: WEN) was cut to Underweight from Neutral at JPMorgan, which trimmed the target price for the fast-food favorite to $6 from $7. Initiations: BioMarin Pharmaceutical (NASDAQ: BMRN) was reinstated with a Neutral rating at Goldman Sachs, which has a $69 target price. BridgeBio Oncology Therapeutics (NASDAQ: BBOT) was started with a Buy rating at Canaccord, with a $23 price target. Coherus Oncology (NASDAQ: CHRS) was started with a Buy rating at Guggenheim, with a $12 target price. Klarna Group (NYSE: KLAR) was initiated with a Hold rating at TD Cowen, and has a $16 target price for the shares. Oklo (NYSE: OKLO) was started with a Neutral rating at JPMorgan, which has set an $83 target for the stock.
ENERGY 3 study met 1 of 2 co-primary endpoints in children with ENPP1 deficiency Treatment with BMN 401 led to statistically significant increases in plasma inorganic pyrophosphate (PPi) concentration, one of the study's co-primary endpoints; however, no improvement was observed in Radiographic Global Impression of Change (RGI-C) scores, the study's other co-primary endpoint and an important clinical measure of change in rickets severity Company is evaluating data to determine next steps SAN RAFAEL, Calif., May 18, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced results from the pivotal Phase 3 ENERGY 3 trial evaluating BMN 401 in children aged 1-12 with ENPP1 deficiency, a rare, serious and progressive genetic condition.
CompaniesMay 18 (Reuters) - BioMarin Pharmaceutical (BMRN.O), opens new tab said on Monday its experimental treatment for a rare genetic condition met one of the two main goals in a late-stage study.
The company was testing its enzyme replacement therapy called BMN 401 in children aged 1 to 12 years with ENPP1 deficiency, a rare, lifelong genetic condition.
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The condition is caused by changes in the ENPP1 gene that result in a decrease in plasma inorganic pyrophosphate, leading to damage to blood vessels, soft tissues and bones.
BioMarin said the therapy met a main goal of significant increases in plasma pyrophosphate through 52 weeks, compared with conventional therapy.
However, it did not show an improvement in a measure of the treatment impact in children with rickets, which causes weak bones, the company said.
Shares of the company were down 2.3% at $50.62.
"We interpret the result as a meaningful clinical failure for BMN 401," H.C. Wainwright analyst Mitchell Kapoor said.
Children with ENPP1 deficiency typically develop a type of rickets that may cause pain and difficulty with movement.
"We are disappointed that the significant increases in plasma PPi observed with BMN 401 did not translate into meaningful clinical improvements for children with ENPP1 deficiency," said Greg Friberg, chief research and development officer at BioMarin.
BioMarin also said it saw no meaningful improvement in rickets severity, which reflects bone weakness and deformities, or in growth, including height and weight.
The company said it is evaluating the data to determine next steps.
The result "materially lowers the probability that BMN 401 becomes a meaningful near-term commercial asset," Kapoor said.
He added that it "increases pressure on BioMarin to deliver elsewhere, including Voxzogo expansion in hypochondroplasia, BMN 333’s long-acting CNP program, and additional business development."
Currently there are no approved treatments for ENPP1 deficiency, and current care focuses on managing symptoms such as bone deformities, pain and movement issues.
Reporting by Sneha S K and Kunal Das in Bengaluru; Editing by Shreya Biswas
Our Standards: The Thomson Reuters Trust Principles., opens new tab
Pre-Market Stock Futures: Futures are trading lower on Tuesday as the sell-off in technology stocks carried through to Monday and is headed down that road today. All of the major indices, except the Dow Jones Industrial Average, which closed 0.32% higher at 49,668, finished the day lower. The small-cap Russell 2000 was the big loser on Monday, closing down 0.63% at 2,775, while the tech-heavy Nasdaq closed lower by 0.51% at 26,090. The S&P 500, which made numerous new highs last week, was last seen at 7,403, down 0.07%. The same issues combined to create the weakness on Monday: worries about higher inflation, the ongoing war in Iran, where President Trump said he called off an imminent attack, and, of course, rising bond yields.
Treasury Bonds: After a brutal beatdown last week, yields across the Treasury curve closed modestly lower as some buyers came in to examine the wreckage. With Wall Street legend Ed Yardeni boldly stating that the bond vigilantes will push yields higher if new Fed Chair Kevin Warsh doesn’t raise rates to combat mounting inflation at some point, the proverbial line in the sand has clearly been drawn. The 30-year bond closed the day at 5.13%, unchanged, and the benchmark 10-year note at 4.59%, also unchanged from Friday.
Oil and Gas: For the first time in over a week, pricing across the energy complex was flat to down, and one thing is for sure. The pressure is mounting on President Trump to wrap up the situation in Iran and reopen the Strait of Hormuz for energy transit. When the dust finally settled Monday, Brent Crude closed the day almot 1% at $108.20, while West Texas Intermediate was marginally higher at $101.30. Natural gas, which has been strong recently, closed the session at $3.02, up 2.13%, as the United States LNG production and sales are quickly becoming the backbone of the world’s gas supply.
Gold: After a rough end to last week, precious metals trended higher on Monday as investors bought into the recent weakness. While the same issues that have muddied the water for almost every asset class since the start of the war with Iran, gold and silver have started to put in a solid base at current trading levels, and could be poised for big moves higher when the Iran issues are resolved. The final trade for Gold was reported at $4,561, up 0.50%, while Silver was last seen at $77.40, up 2.06%.
Crypto: Cryptocurrencies declined on Monday amid a broad sell-off, with Bitcoin sliding to a two-week low near $76,400. The drop triggered more than $660 million in liquidations across the crypto market, as rising bond yields, persistent inflation, and geopolitical tensions weighed on investor risk appetite. It confirms what many have been saying about the crypto market for months: most upticks and positive days are likely mostly short covering. At 8 AM EDT, Bitcoin was trading at $76,680, while Ethereum was quoted at $2,111.
24/7 Wall St. reviews dozens of analyst research reports daily to identify new investment ideas for both investors and traders. Some of these daily analyst calls cover stocks to buy. Other calls cover stocks to sell or avoid. Remember that no single analyst call should ever be used as a basis to buy or sell a stock.
Here are some of the top Wall Street analyst upgrades, downgrades, and initiations seen on Tuesday, May 19, 2026.
Upgrades: American Tower (NYSE: AMT | AMT Price Prediction) was upgraded to Outperform from Market Perform at Bernstein, which has a $207 target price for the shares. Assured Guaranty (NYSE: AGO) was upgraded to Buy from Neutral at UBS, with a $94 target price. Credicorp (NYSE: BAP) was raised to Buy from Hold at HSBC, with the target price for the stock bumped to $350 from $320. Jazz Pharmaceuticals (NASDAQ: JAZZ) was raised to Buy from Neutral at UBS, which launched the target price for the stock to $307 from $188. Stubhub Holdings (NYSE: STUB) was upgraded to Buy from Neutral at Guggenheim, which lifted the target price for the share to $12.50 from $8.50. Downgrades: Bank of America (NYSE: BAC) was downgraded to Hold from Buy at CFRA, without a target price. Citigroup (NYSE: C) was also cut to Hold from Buy at CFRA, without a target price. CrowdStrike Holdings (NASDAQ: CRWD) was double downgraded to Sell from Buy at DZ Bank, with a $500 target price. Fortinet (NASDAQ: FTNT) was also double downgraded to Sell from Buy at DZ Bank, with a $125 target price. Hanover Insurance Group (NYSE: THG) was downgraded to Market Perform from Outperform at BMO Capital, which bumped the target price for the stock to $203 from $194. Initiations: Alnylam Pharmaceuticals (NASDAQ: ALNY) was initiated with a Buy rating at Citigroup, which has set a $380 price target for the shares. BioMarin Pharmaceutical (NASDAQ: BMRN) was initiated with a Buy rating at Citigroup with a $75 target price. Cemex SAB (NYSE: CX) was assumed with a Neutral rating at Grupo Santander with a $14 target price. X-Energy (NASDAQ: XE) was started with a Buy rating at UBS, with a $40 target. JPMorgan has an Overweight rating for the stock with a $38 target, while UBS has a Buy rating and a nd a $40 target price. The stock was a recent successful IPO, backed by Amazon and Ken Griffin from Citadel. Zeta Global Holdings (NYSE: ZETA) was initiated with a Buy rating at Bank of America, with a $24 target price objective.
Key Takeaways BioMarin shares fell 4% after BMN 401 missed one of two main goals in the phase III ENERGY 3 study.BMRN reported higher plasma PPi levels, but no improvement in radiographic scores after 52 weeks.BMN 401 also missed secondary goals tied to rickets severity, height and weight growth measures. Shares of BioMarin Pharmaceutical (BMRN - Free Report) were down 4% on Monday after it reported results from the phase III ENERGY 3 study, which evaluated BMN 401, an investigational enzyme replacement therapy (ERT), for a rare genetic disorder called ENPP1 deficiency in children aged 1 to 12.
The study did not meet one of its two main goals.
BMRN’s Stock PerformanceThe mixed results were a setback for BioMarin, which added BMN 401 through its $270 million acquisition of Inozyme Pharma last year. This ERT was the lead asset in Inozyme’s pipeline, and the ENERGY 3 study was already underway at the time of the acquisition.
Year to date, the stock has lost 16% compared with the industry’s 3% fall.
Image Source: Zacks Investment Research
More on BioMarin’s ENERGY 3 Study ResultsThe study assessed two primary endpoints — changes in plasma inorganic pyrophosphate (PPi) and Radiographic Global Impression of Change (RGI-C) scores after 52 weeks of treatment. While treatment with BMN 401 after 52 weeks resulted in statistically significant increases in plasma PPi, there was no corresponding improvement in RGI-C scores.
An increase in plasma PPi is considered an important biomarker response because ENPP1 deficiency leads to low PPi levels, which can result in progressive damage to blood vessels, soft tissues and bones.
However, BMN 401 failed to demonstrate improvement in RGI-C scores, a key measure used to assess treatment impact in children with rickets. The lack of radiographic improvement suggests that the biomarker gains did not translate into measurable skeletal benefits during the study period.
The therapy also did not show positive trends across secondary endpoints, including Rickets Severity Score (RSS), a measure of rickets severity, and growth Z-scores evaluating height/body length and weight.
BioMarin stated that it will continue analyzing the complete ENERGY 3 dataset and engage with global regulatory authorities to determine the next steps for BMN 401’s development program. The company also plans to present detailed findings from the study at a future medical meeting.
BMRN’s Zacks RankThe stock currently carries a Zacks Rank #3 (Hold).
Key Picks Among Biotech StocksSome better-ranked stocks from the sector are Immunocore (IMCR - Free Report) and Indivior Pharmaceuticals (INDV - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, estimates for Immunocore’s 2026 loss per share have narrowed from 97 cents to 16 cents. Over the same period, estimates for 2027 have improved from a loss of 39 cents to earnings of 11 cents. IMCR shares have lost 18% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters but missed the mark on one occasion, delivering an average surprise of 46.66%.
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Study met primary endpoint, exceeding expectations with a highly statistically significant improvement in annualized growth velocity (AGV, change from baseline) of 2.33 cm/yr compared to placebo at week 52, as well as statistically significant increases in standing height and height Z-score
Study demonstrated statistically significant improvement in arm span at week 52 compared to placebo, a key measure linked to functional independence
Safety findings consistent with the established profile in achondroplasia with no new safety signals identified
Supplemental New Drug Application (sNDA) submission to U.S. Food and Drug Administration (FDA) planned for third quarter, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities
Full data to be presented at an upcoming medical meeting
, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualized growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p<0.0001). Children who received VOXZOGO also showed a statistically significant increase in standing height (p<0.0001) and height Z-score (p<0.0001) versus placebo after one year of treatment.
Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study. These positive findings could have important implications for children with hypochondroplasia, offering improvements in reach, daily activities and independence, which have been highlighted as meaningful outcomes by the community.
"As someone who treats children with hypochondroplasia, I find these positive results tremendously encouraging. Seeing these improvements in growth is a milestone we have hoped for after so many years without treatment options," said Dr. Andrew Dauber, who is the lead study investigator and Chief of Endocrinology at Children's National Hospital in Washington, D.C. "These data suggest we may be approaching a new era in how we care for children with hypochondroplasia."
"For the first time, a pivotal Phase 3 study in hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span, highlighting VOXZOGO's potential to fundamentally change the treatment landscape for this condition," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "We are incredibly pleased with these results, which exceeded our expectations for this study, and we are deeply grateful to the families and investigators who made this outcome possible. We look forward to sharing these data with regulatory authorities and the broader community as we work to bring this medicine to children living with hypochondroplasia around the world."
The safety findings in the study for VOXZOGO were consistent with the established profile in achondroplasia and no new safety signals were observed. Full results from the CANOPY-HCH-3 study will be presented at an upcoming medical meeting.
The supplemental New Drug Application (sNDA) submission to the U.S. Food and Drug Administration (FDA) is planned for the third quarter of 2026, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities.
About CANOPY-HCH-3
CANOPY-HCH-3 (Study 111-303) is a global Phase 3, 1:1 randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of VOXZOGO in 80 children ages 3 to 17 with hypochondroplasia. The primary endpoint of the study was change from baseline in annualized growth velocity (AGV) at 52 weeks compared to placebo. Key secondary pre-specified endpoints included changes from baseline in standing height, height Z-score, arm span, upper to lower body segment ratio, and health-related quality of life, along with assessments of safety and tolerability. Study participants will continue to be followed in a long-term extension study.
About Hypochondroplasia
Hypochondroplasia is a rare, genetic skeletal dysplasia characterized by impaired bone growth, leading to disproportionate short stature and skeletal differences that can affect the long bones, spine, and other parts of the skeleton and may impact physical functioning and overall quality of life. The condition presents with a broad and variable clinical spectrum and may include otolaryngologic (related to the ears, nose and throat) and neurological complications and is often diagnosed in toddlerhood or early school age based on clinical and radiological findings.
There are currently no medicines approved by the U.S. Food and Drug Administration or the European Medicines Agency for the treatment of hypochondroplasia.
For more information about our clinical trials in hypochondroplasia, achondroplasia and other skeletal conditions, please visit clinicaltrials.biomarin.com.
About VOXZOGO (vosoritide)
VOXZOGO is approved in the U.S., Japan and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses, and VOXZOGO is indicated in the EU for the treatment of achondroplasia in children 4 months of age and older whose epiphyses are not closed, as confirmed by appropriate genetic testing. In the U.S., this indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin submitted its U.S. supplemental New Drug Application (sNDA) for full approval of VOXZOGO for achondroplasia in April 2026, and the company expects to be notified of sNDA acceptance by the third quarter of 2026.
The use of VOXZOGO to treat hypochondroplasia has not yet been approved by any regulatory agency.
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO?
VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO?
The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken?
VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO?
Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see additional safety information in the full Prescribing Information and Patient Information.
About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with a portfolio of commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the development of BioMarin's VOXZOGO program generally, and the results of the pivotal Phase 3 CANOPY-HCH-3 study evaluating treatment with VOXZOGO in children with hypochondroplasia particularly; the safety profile and potential benefits of VOXZOGO for children with hypochondroplasia, including VOXZOGO's potential to fundamentally change the treatment landscape for hypochondroplasia; BioMarin's plans to submit for regulatory approval to global health authorities in the second half of 2026; and BioMarin's expectations regarding the demand for VOXZOGO. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, the final analysis of the results from CANOPY-HCH-3, any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
