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.
Further Reading Five stocks we like better than BioMarin Pharmaceutical Could Truth API Become Trump Media’s First Meaningful Revenue Driver? Small Caps Are Crushing the S&P 500—3 Stocks Still Worth Buying Moog Is More Than a Missile Maker, and Wall Street Is Noticing A Boring Dividend Growth Strategy Becomes a Solid Defensive Play
Receive News & Ratings for BioMarin Pharmaceutical Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for BioMarin Pharmaceutical and related companies with MarketBeat.com's FREE daily email newsletter.
« PREVIOUS HEADLINEB&D White Capital Company LLC Raises Stock Holdings in Avantis U.S. Small Cap Equity ETF $AVSC
NEXT HEADLINE »Fifth Third Bancorp Purchases 28,699 Shares of Prestige Consumer Healthcare Inc. $PBH
Analyst’s Disclosure: I/we have no stock, option or similar derivative position in any of the companies mentioned, and no plans to initiate any such positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.
Seeking Alpha's Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. Any views or opinions expressed above may not reflect those of Seeking Alpha as a whole. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank. Our analysts are third party authors that include both professional investors and individual investors who may not be licensed or certified by any institute or regulatory body.
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.
Recommended Stories Five stocks we like better than BioMarin Pharmaceutical The Ugliest Stocks in the Market Just Got a Very Expensive Vote of Confidence Is Domino’s Stock Serving Up a Buying Opportunity? A $1T Black Hole: SpaceX Eyes Pentagon AI to Break Free Why Gold Miners Could Be the Market’s Biggest Comeback Story Want to see what other hedge funds are holding BMRN? Visit HoldingsChannel.com to get the latest 13F filings and insider trades for BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report).
Receive News & Ratings for BioMarin Pharmaceutical Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for BioMarin Pharmaceutical and related companies with MarketBeat.com's FREE daily email newsletter.
« PREVIOUS HEADLINEBank of New York Mellon Corp Has $111.65 Million Stake in F5, Inc. $FFIV
NEXT HEADLINE »Bessemer Group Inc. Has $6 Million Stock Position in Starbucks Corporation $SBUX
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.
For new and old investors, taking full advantage of the stock market and investing with confidence are common goals. Zacks Premium provides lots of different ways to do both.
The popular research service can help you become a smarter, more self-assured investor, giving you access to daily updates of the Zacks Rank and Zacks Industry Rank, the Zacks #1 Rank List, Equity Research reports, and Premium stock screens.
Zacks Premium also includes the Zacks Style Scores.
What are the Zacks Style Scores? The Zacks Style Scores is a unique set of guidelines that rates stocks based on three popular investing types, and were developed as complementary indicators for the Zacks Rank. This combination helps investors choose securities with the highest chances of beating the market over the next 30 days.
Each stock is assigned a rating of A, B, C, D, or F based on their value, growth, and momentum characteristics. Just like in school, an A is better than a B, a B is better than a C, and so on -- that means the better the score, the better chance the stock will outperform.
The Style Scores are broken down into four categories:
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.
Growth ScoreGrowth investors, on the other hand, are more concerned with a company's financial strength and health, and its future outlook. The Growth Style Score examines things like projected and historic earnings, sales, and cash flow to find stocks that will experience sustainable growth over time.
Momentum ScoreMomentum trading is all about taking advantage of upward or downward trends in a stock's price or earnings outlook, and these investors live by the saying "the trend is your friend." The Momentum Style Score can pinpoint good times to build a position in a stock, using factors like one-week price change and the monthly percentage change in earnings estimates.
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 is a proprietary stock-rating model that harnesses the power of earnings estimate revisions, or changes to a company's earnings expectations, to help investors build a successful portfolio.
#1 (Strong Buy) stocks have produced an unmatched +23.94% average annual return since 1988, which is more than double the S&P 500's performance over the same time frame. However, the Zacks Rank examines a ton of stocks, and there can be more than 200 companies with a Strong Buy rank, and another 600 with a #2 (Buy) rank, on any given day.
But it can feel overwhelming to pick the right stocks for you and your investing goals with over 800 top-rated stocks to choose from.
That's where the Style Scores come in.
To have the best chance of big returns, you'll want to always consider stocks with a Zacks Rank #1 or #2 that also have Style Scores of A or B, which will give you the highest probability of success. If you're looking at stocks with a #3 (Hold) rank, it's important they have Scores of A or B as well to ensure as much upside potential as possible.
As mentioned above, the Scores are designed to work with the Zacks Rank, so any change to a company's earnings outlook should be a deciding factor when picking which stocks to buy.
A stock with a #4 (Sell) or #5 (Strong Sell) rating, for instance, even one with Scores of A and B, will still have a declining earnings forecast, and a greater chance its share price will fall 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 #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.
Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here.
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%.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 EPS have increased from $3.10 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.69 from $3.47. INDV shares have risen 2% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 65.44%.
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.
CompaniesMay 20 (Reuters) - BioMarin Pharmaceutical (BMRN.O), opens new tab said on Wednesday its treatment for a rare condition that leads to short stature helped boost growth in children, meeting the main goal of a late-stage study and sending its shares up nearly 5% in extended trading.
Here are some details:
Keep up with the latest medical breakthroughs and healthcare trends with the Reuters Health Rounds newsletter. Sign up here.
The drug, Voxzogo, significantly increased the annualized growth rate in patients with hypochondroplasia after 52 weeks compared with placebo, with treated patients growing 2.33 cm more, the company said.
Hypochondroplasia is a rare genetic skeletal disorder that affects bone growth and leads to short-limbed dwarfism.
Patients receiving the therapy saw significant improvement in standing height and arm span, a key secondary goal of the study.
The 80-patient study evaluated the drug in children aged 3 to 17 years.
There are currently no approved treatments for the disorder, BioMarin said.
Jefferies analyst Andrew Tsai said strong late-stage data for BioMarin's Voxzogo supports the underlying growth biology and could de-risk BridgeBio's <BBIO.O> oral rival infigratinib, which expects a study readout in the second half of 2026.
"Our $1 billion peak sales estimate in achondroplasia/hypochondroplasia could be conservative," Tsai added.
Voxzogo was already approved in 2021 in the U.S. for patients with achondroplasia, a form of dwarfism.
BioMarin plans to submit a supplemental application to U.S. regulators in the third quarter of this year.
Reporting by Siddhi Mahatole in Bengaluru; Editing by Diti Pujara
Our Standards: The Thomson Reuters Trust Principles., opens new tab
Hypochondroplasia is a form of genetic, short-limbed dwarfism.
The study met its primary endpoint, showing a statistically significant improvement in annualized growth velocity compared to placebo, which has excited investors and analysts alike.
VOXZOGO Study Meets Primary EndpointThe CANOPY-HCH-3 study demonstrated a 2.33 cm/year increase in annualized growth velocity at week 52, significantly exceeding expectations.
With plans for a supplemental New Drug Application submission to the FDA in the third quarter of 2026, these results could pave the way for new treatment options in a market currently lacking approved therapies for hypochondroplasia.
Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study.
“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, EVP and Chief Research & Development Officer at BioMarin
Analyst Consensus & Recent Actions: The stock carries a Buy rating with an average price target of $87.47. Recent analyst moves include:
B of A Securities: Buy (Lowers Target to $80.00) (May 19) Citigroup: Initiated with Buy (Target $75.00) (May 19) HC Wainwright & Co.: Neutral (Lowers Target to $50.00) (May 18) How BioMarin Ranks On Value And MomentumBelow is the Benzinga Edge scorecard for BioMarin Pharmaceuticals, highlighting its strengths and weaknesses compared to the broader market:
Value: 67.93 — The stock is performing moderately well in terms of value metrics. Momentum: 12.86 — Stock is underperforming the broader market. BMRN Price Action: BioMarin Pharmaceutical shares were up 8.05% at $54.24 at the time of publication on Thursday, according to Benzinga Pro data.
Photo: Shutterstock
Market News and Data brought to you by Benzinga APIs
Key Takeaways BioMarin's Voxzogo boosted annualized growth velocity by 2.33 cm/yr versus placebo.BMRN plans a U.S. filing in Q3 2026 to expand Voxzogo into hypochondroplasia.Voxzogo faces new achondroplasia rivals as BioMarin targets a new growth opportunity. Shares of BioMarin Pharmaceutical (BMRN - Free Report) were up 3% in pre-market trading today after the company reported positive top-line results from the phase III CANOPY-HCH-3 study evaluating Voxzogo for label expansion in hypochondroplasia.
The study met its primary endpoint, with patients treated with the drug for 52 weeks showing a statistically significant increase in annualized growth velocity (AGV) of 2.33 cm/yr compared to placebo. Voxzogo also showed significant improvement across several key secondary pre-specified endpoints, including standing height, height Z-score and arm span.
Voxzogo was approved by the FDA in 2021 to treat children of all ages with achondroplasia, the most common form of dwarfism. It is approved for similar indications in Europe, Japan and Australia.
Hypochondroplasia is a rare genetic skeletal growth disorder marked by short stature and impaired bone growth. The condition is generally considered a milder form of achondroplasia. Per BioMarin, there are currently no therapies approved by the FDA or the EMA for hypochondroplasia, highlighting a significant unmet medical need.
Data from the CANOPY-HCH-3 study will support regulatory submissions seeking label expansion for Voxzogo in hypochondroplasia. BioMarin expects to submit a regulatory filing in the United States in the third quarter of 2026, followed by submissions to the EMA and other global regulatory authorities. If approved, the label expansion could broaden the drug’s addressable market opportunity.
Since 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.
BMRN’s Stock PerformanceYear to date, shares of BioMarin have lost nearly 16% compared with the industry’s 3% fall.
Image Source: Zacks Investment Research
BMRN’s Voxzogo Faces an Evolving Competitive LandscapeThe 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 this approval, Voxzogo was the only FDA-approved therapy for the condition. The approval of ASND’s Yuviwel was also a key factor behind BioMarin withdrawing its prior 2027 revenue guidance of approximately $4 billion.
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.
Against this increasingly competitive backdrop, potential expansion of Voxzogo into hypochondroplasia could provide BioMarin with an additional growth avenue and help offset future competitive pressure in the achondroplasia market. With no FDA- or EMA-approved therapies currently available for hypochondroplasia, the company may also benefit from a potential first-mover advantage in the indication.
BMRN’s Zacks RankThe stock currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
A month has gone by since the last earnings report for BioMarin Pharmaceutical (BMRN - Free Report) . Shares have added about 0.2% in that time frame, underperforming the S&P 500.
Will the recent positive trend continue leading up to its next earnings release, or is BioMarin due for a pullback? Well, first let's take a quick look at the latest earnings report in order to get a better handle on the recent drivers for BioMarin Pharmaceutical Inc. before we dive into how investors and analysts have reacted as of late.
Q1 Earnings Miss, Sales Beat EstimatesBioMarin 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.
Quarter in DetailNet 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 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.
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.
Kuvan revenues declined 4% to $24 million due to generic competition.
2026 GuidanceIn April 2026, BioMarin announced that it completed the acquisition of Amicus Therapeutics for $4.8 billion. With this transaction, the company 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.
How Have Estimates Been Moving Since Then?In the past month, investors have witnessed a downward trend in estimates revision.
The consensus estimate has shifted -25.57% due to these changes.
VGM ScoresAt this time, BioMarin has a nice Growth Score of B, though it is lagging a lot on the Momentum Score front with an F. However, the stock was allocated a score of A on the value side, putting it in the top 20% for value investors.
Overall, the stock has an aggregate VGM Score of B. If you aren't focused on one strategy, this score is the one you should be interested in.
OutlookEstimates have been broadly trending downward for the stock, and the magnitude of these revisions looks promising. Notably, BioMarin has a Zacks Rank #3 (Hold). We expect an in-line return from the stock in the next few months.
Performance of an Industry PlayerBioMarin belongs to the Zacks Medical - Biomedical and Genetics industry. Another stock from the same industry, Illumina (ILMN - Free Report) , has gained 16.9% over the past month. More than a month has passed since the company reported results for the quarter ended March 2026.
Illumina reported revenues of $1.09 billion in the last reported quarter, representing a year-over-year change of +4.8%. EPS of $1.15 for the same period compares with $0.97 a year ago.
For the current quarter, Illumina is expected to post earnings of $1.24 per share, indicating a change of +4.2% from the year-ago quarter. The Zacks Consensus Estimate remained unchanged over the last 30 days.
The overall direction and magnitude of estimate revisions translate into a Zacks Rank #3 (Hold) for Illumina. Also, the stock has a VGM Score of C.
