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Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) in late-onset Pompe disease (LOPD) were presented at the 31st Annual Congress of the World Muscle Society (WMS), in Hiroshima, Japan.

These results from the PROPEL open-label extension (OLE) study of POMBILITI + OPFOLDA, which were shared in a late-breaking presentation, demonstrated durable motor function outcomes and relative stabilization of pulmonary function over long-term follow-up. The analysis included data from 82 individuals who received POMBILITI + OPFOLDA continuously from baseline observed in the initial PROPEL study (PROPEL baseline) to year five. For the enzyme replacement therapy (ERT)-experienced cohort, the mean duration of prior ERT at baseline was 7.6 years. These data represent long-term benefits for people with LOPD followed in the study, including both ERT-experienced and ERT-naïve cohorts. 

"These five-year results demonstrate that POMBILITI and OPFOLDA can provide durable, long-term benefits for people living with late-onset Pompe disease, including those who had been on enzyme replacement therapy for years before enrolling in the study," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Sustaining motor function and limiting pulmonary decline for up to five years in a progressive disease like LOPD is clinically meaningful, and we remain committed to continuing to share long-term evidence to inform the care of people with this rare genetic condition."

Table 1. Efficacy Measures With POMBILITI + OPFOLDA From PROPEL Baseline (BL) for up to Five Years

ERT-Experienced

to Year 5
(n=62 at BL)

ERT-Naïve

to Year 4.5

(n=20 at BL)

Mean (standard deviation,
SD) change from baseline
in % predicted six-minute
walk distance (pp6MWD)     

0.7% (7.76)

10.5% (11.44)

Mean (SD) change from
baseline in % predicted
forced vital capacity
(ppFVC)

-2.8% (8.55)

-4.4% (6.89)

No new safety signals were identified. 51.2% of participants (n=42) experienced treatment-related adverse events, the majority of which were manageable. Five individuals discontinued treatment for safety reasons, including two due to serious treatment-related adverse events (chills and hypoxia [n=1], anaphylactoid reaction [n=1]).

Phase 1/2 Data Show Positive Dystrophin Expression and Favorable Tolerability With Nivudirsen (BMN 351)

Additional presentations at WMS included data evaluating nivudirsen (BMN 351), an investigational antisense oligonucleotide for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Included in these presentations was an update from the ongoing Phase 1/2 351-201 study, which was previously shared at the 19th International Congress on Neuromuscular Diseases in July. Results from 351-201 showed treatment with nivudirsen was generally well tolerated and led to consistently positive outcomes across multiple measures of therapeutic benefit, including expression of near full-length dystrophin.

Below are key presentations at WMS, with all times listed in Japan Standard Time:

Phase 1/2 Results of BMN 351, an Antisense Oligonucleotide for Exon 51 Skip-Amenable Duchenne Muscular Dystrophy
Poster Presentation #2.66P
Wednesday, Sept. 30, 5:15 – 6:15 p.m.

Comprehensive Panel of Pharmacokinetic, Pharmacodynamic and Biomarker Measures Drives Clinical Development of BMN 351 for Duchenne Muscular Dystrophy
E-Poster Presentation #2.43eP
Wednesday, Sept. 30, 5:15 – 6:15 p.m.

Muscle Composition and Muscle Injury Biomarkers Improve With BMN 351 Treatment
E-Poster Presentation #2.59eP
Wednesday, Sept. 30, 5:15 – 6:15 p.m.

Long-Term (Five-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline
E-Poster Presentation #3.081LBeP
Friday, Oct. 2, 2:15 – 3:15 p.m. 

Long-Term (Four-Year) Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease Treated From PROPEL Baseline
Poster Presentation #3.56P
Friday, Oct. 2, 2:15 – 3:15 p.m.

Baseline Disease Burden and Real-World Monitoring Practices: Global Insights From the followME Pompe Journey Registry
Poster Presentation #3.57P
Friday, Oct. 2, 2:15 – 3:15 p.m.

ATB200-03/07 Danish Subpopulation: Four-Year Efficacy and Safety of Cipaglucosidase Alfa Plus Miglustat in Adults With Late-Onset Pompe Disease
E-Poster Presentation #3.35eP
Friday, Oct. 2, 2:15 – 3:15 p.m.

About the PROPEL Open-Label Extension Study

The Phase 3 open-label extension (OLE) study (ATB200-07; NCT04138277) is a multicenter, international study designed to evaluate the long-term safety and efficacy of POMBILITI + OPFOLDA in adults with late-onset Pompe disease (LOPD) who completed the Phase 3 PROPEL study (ATB200-03; NCT03729362). Of the 118 people treated in the OLE, 82 continued POMBILITI + OPFOLDA treatment from PROPEL (62 ERT-experienced prior to PROPEL; 20 ERT-naïve) and 37 switched from the comparator arm to POMBILITI + OPFOLDA (29 ERT-experienced; eight ERT-naïve).

The study evaluates long-term changes from PROPEL baseline in six-minute walk distance (6MWD, the primary endpoint in PROPEL), forced vital capacity (FVC), creatine kinase (CK) and hexose tetrasaccharide (Hex4) levels, and patient-reported outcomes, as well as long-term safety.

About Study 351-201

The 351-201 trial (NCT06280209) is a Phase 1/2 multicenter open-label dose-escalation study evaluating BMN 351 for the treatment of boys aged 4 to 10 with Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping. The primary endpoints examine the safety and tolerability of BMN 351, the secondary endpoints assess plasma, urine and muscle pharmacokinetics, and key exploratory endpoints include exon-skipping efficiency, dystrophin expression, immunogenicity and functional assessments. 

The study is being conducted in three cohorts, with Cohort 1 being divided into Cohort 1A and Cohort 1B. In Cohort 1A, three boys received increasing doses once every two weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351. In part two, the participants in Cohort 1A transitioned to once-weekly dosing. The participants in Cohort 1B, 2 and 3 will receive low (6 mg/kg), medium (9 mg/kg) and high (12 mg/kg) doses of BMN 351 and continue once-weekly dosing at that same dose.

For more information, please visit clinicaltrials.biomarin.com.

About Late-Onset Pompe Disease

Late-onset Pompe disease (LOPD) is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. LOPD can be severe and debilitating with progressive muscle weakness throughout the body that worsens over time, particularly in skeletal muscles and muscles that control breathing.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe neuromuscular condition that leads to progressive muscle weakness and premature mortality. DMD is caused by genetic changes that result in the absence or dysfunction of dystrophin, an essential protein that is necessary for preserving muscle function.

As an X-linked disorder, DMD primarily affects young boys, with onset of symptoms occurring around 2 to 3 years of age. The incidence of DMD is up to 1 in 3,600 to 6,000 live male births.

Individuals with DMD experience serious functional impairments, including loss of limb strength and ambulation, and respiratory and cardiovascular impacts that require lifelong care.

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 is designed to reduce loss of enzyme activity in the blood.

Patient Support Accessing POMBILITI + OPFOLDA

To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-866-906-6100 or email [email protected]. For more information about POMBILITI + OPFOLDA, please visit www.pombilitiopfolda.com. For additional information regarding these products, please contact BioMarin Medical Information at [email protected].

POMBILITI + OPFOLDA U.S. Indication and Important Safety Information

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 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 the 31st Annual Congress of the World Muscle Society (WMS), including the potential benefits and safety of POMBILITI + OPFOLDA in late-onset Pompe disease as well as of nivudirsen (BMN 351) for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping; BioMarin's plans and expectations for the development of POMBILITI + OPFOLDA and nivudirsen; and BioMarin's estimate regarding the incidence of DMD. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the 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, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, BioMarin RareConnections®, POMBILITI® and OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc. or its affiliates.

Contacts:

Investors

Media

Traci McCarty

Katherine Powell

BioMarin Pharmaceutical Inc.     

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 827-2968

SOURCE BioMarin Pharmaceutical Inc.

Key Takeaways BMRN stops Voxzogo development for Noonan syndrome, citing study feasibility and the treatment landscape.Voxzogo generated $472 million in first-half 2026 sales, up 9% year over year on strong demand.BMRN expects $1.00-$1.05 billion in 2026 Voxzogo sales, with hypochondroplasia approval targeted for 2027. BioMarin Pharmaceutical (BMRN - Free Report) announced that it is discontinuing the development of Voxzogo (vosoritide) for children with Noonan syndrome, a rare genetic condition that can affect growth and development in multiple parts of the body.

This announcement came via an SEC filing on Tuesday, which attributed the decision to the study’s feasibility and the current treatment landscape. The company clarified that the decision was not related to the safety or efficacy of Voxzogo.

In March, BioMarin ended development of the drug for three other short stature indications, Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency. Unlike the Noonan syndrome, these studies were stopped due to the occurrence of an adverse event called slipped capital femoral epiphysis (SCFE) in study participants across two investigator-sponsored studies. At the time, BioMarin stated that it would continue studying Voxzogo for Noonan syndrome.

Voxzogo was approved by the FDA in 2021 as the first approved treatment for achondroplasia, the most common form of dwarfism. The drug is a C-type natriuretic peptide (CNP) analog administered once daily as a subcutaneous injection. CNP is a naturally occurring signaling molecule that plays a role in regulating bone growth.

BMRN Stock PerformanceYear to date, shares of the company have gained 8.5% compared with the industry’s 5.5% growth.

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Voxzogo: A Key Driver of BioMarin’s ToplineVoxzogo has seen rapid uptake since launch. The drug has generated $472 million in sales during the first half of 2026, up 9% year over year, driven by strong prescription demand. Voxzogo is currently BioMarin's largest product by net product revenues.

Management expects sales in the second half of the year to exceed those in the first half. It expects to generate $1.00-$1.05 billion from Voxzogo sales in 2026, putting the drug on track to achieve blockbuster status.

Despite the discontinuation of development for Noonan syndrome, Voxzogo retains other potential label-expansion opportunities. BioMarin submitted a regulatory filing seeking label expansion for hypochondroplasia, which is generally considered to be a milder form of achondroplasia. This filing is supported by data from the phase III CANOPY-HCH-3 study, which met its primary and key secondary endpoints. If approved, BioMarin expects a potential launch in 2027.

BioMarin is also advancing Voxzogo in a label expansion study for another short-stature pathway condition called idiopathic short stature (without ACAN-deficiency).

BioMarin Confronts a Changing Competitive Landscape for VoxzogoVoxzogo faces increasing competition from Ascendis Pharma (ASND - Free Report) , whose Yuviwel (navepegritide) received FDA approval for achondroplasia in February 2026. Like Voxzogo, Yuviwel is a CNP therapy but is administered once weekly. The ASND drug is designed to provide continuous exposure to active CNP, potentially offering a more convenient dosing profile than the BMRN drug.

BioMarin had previously alleged that Yuviwel infringed its intellectual property covering CNP technology, triggering patent disputes across the United States, Brazil, Denmark, Germany and South Korea, including before the U.S. International Trade Commission. Recently, the two companies entered into a settlement that meaningfully changed the economics of competition.

Per the settlement terms, BioMarin will grant Ascendis a non-exclusive, worldwide license to certain patents covering Yuviwel and related products, allowing Ascendis to research, develop, manufacture and commercialize the drug across current and potential indications.

In return, Ascendis will pay BioMarin royalties equal to 20% of annual net sales in the United States and 18% in the European Union, Brazil and South Korea, from the first commercial sale in each territory through May 20, 2030. The U.S. royalty is also applied retroactively to Yuviwel's first commercial sale. All pending lawsuits between the two companies will also be dismissed.

The settlement provides BioMarin with a meaningful royalty stream from a product that competes directly with Voxzogo. However, the royalties do not eliminate the competitive risk. If Yuviwel takes substantial market share from Voxzogo, BioMarin could lose Voxzogo revenues while gaining royalty income on Ascendis' sales. The net economic impact will therefore depend on the pace of Yuviwel adoption, the extent to which it expands the overall treated population and how much of its sales come at Voxzogo's expense.

BioMarin’s Voxzogo could have another potential competitor as early as next year. Recently, BridgeBio Pharma (BBIO - Free Report) submitted a regulatory filing with the FDA for infigratinib, an FGFR3 inhibitor that could become the first approved oral therapy for achondroplasia. The company expects a U.S. launch in mid-2027, subject to approval.

BMRN’s Zacks RankBioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Key Takeaways BioMarin will receive royalties on Yuviwel sales, turning a patent dispute into a royalty stream.BMRN gets 20% U.S. royalties and 18% in the EU, Brazil and South Korea through May 2030.Yuviwel reached 8 million euros in second-quarter revenue and enrolled more than 220 patients by July 31. For BioMarin Pharmaceutical (BMRN - Free Report) , the FDA approval of Ascendis Pharma’s (ASND - Free Report) Yuviwel in February had created a problem — a new entrant targeting the same achondroplasia market as Voxzogo, its key revenue driver. But a recent settlement between the two companies has changed the economics of that competition in a meaningful way.

Rather than continue fighting a competitor that had already entered the market, BioMarin has found a way to make the competitor pay it. Under the settlement, Ascendis will pay BioMarin royalties on Yuviwel sales in the United States, European Union, Brazil and South Korea, effectively giving BMRN an economic interest in a drug that could otherwise take share from Voxzogo.

BMRN Turns a Patent Dispute Into a Royalty StreamWhen Voxzogo secured FDA approval in 2021, it became the first FDA-approved therapy for achondroplasia, the most common form of dwarfism. The drug is a C-type natriuretic peptide (CNP) analog administered once daily as a subcutaneous injection. CNP is a naturally occurring signaling molecule involved in bone growth.

When Ascendis launched Yuviwel in April, its entry was a significant challenge for BioMarin, as Voxzogo had previously been the only approved treatment for achondroplasia. Like Voxzogo, Yuviwel is also a CNP therapy but is administered once weekly. The ASND drug is designed to provide continuous exposure to active CNP, potentially offering a more convenient dosing profile than the BMRN drug.

BioMarin had alleged that Yuviwel infringed its intellectual property covering CNP technology, triggering patent disputes across the United States, Brazil, Denmark, Germany and South Korea, including before the U.S. International Trade Commission. But instead of continuing to fight, both companies have settled their disputes with a licensing deal.

Per the settlement terms, BioMarin will grant Ascendis a non-exclusive, worldwide license to certain patents covering Yuviwel and related products, allowing Ascendis to research, develop, manufacture and commercialize the drug across current and potential indications.

In return, Ascendis will pay BioMarin royalties equal to 20% of annual net sales in the United States and 18% in the European Union, Brazil and South Korea, from the first commercial sale in each territory through May 20, 2030. The U.S. royalty is also applied retroactively to Yuviwel's first commercial sale. All pending lawsuits between the two companies will also be dismissed.

Competitive Threat for BMRN Still RemainsThe settlement changes the competitive equation for BioMarin. Previously, every dollar of Yuviwel sales represented a potential dollar of revenue at risk for Voxzogo. Under the new agreement, Yuviwel sales can also generate revenues for BioMarin through royalties. This means that while greater adoption of Yuviwel could still put pressure on Voxzogo, BMRN will now participate in the growth of its competitor.

The opportunity is becoming more meaningful as Yuviwel gains traction. Ascendis generated €8 million in Yuviwel revenues in the second quarter of 2026, its first quarter of launch, and had enrolled more than 220 patients through July 31. More than 100 healthcare providers had prescribed the drug, with more than 65% of enrollments approved for reimbursement. Ascendis is also preparing for further expansion, with its EU filings under review and additional studies underway.

Still, the settlement does not eliminate the competitive risk for BioMarin. Royalties are only a partial offset to any erosion in Voxzogo sales, and the economics will ultimately depend on how quickly Yuviwel penetrates the market and how much of that growth comes at Voxzogo’s expense.

BioMarin’s Voxzogo could have another potential competitor by this time next year. Recently, BridgeBio Pharma (BBIO - Free Report) has submitted a regulatory filing with the FDA for infigratinib, an FGFR3 inhibitor that could become the first approved oral therapy for achondroplasia. The company expects a U.S. launch in mid-2027, subject to approval.

The arrival of another potentially differentiated therapy underscores that BioMarin’s competitive challenge is unlikely to end with Yuviwel. The company may have found a clever way to monetize one threat, but it will still need to defend Voxzogo as the achondroplasia market becomes increasingly competitive.

BMRN’s Price Performance, Valuation & EstimatesShares of BioMarin have risen 13% year to date, significantly outperforming the industry’s 11% growth.

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From a valuation standpoint, the company is currently trading at a premium to the industry. Based on the price-to-sales (P/S) ratio, the stock trades at 3.06 times forward 12-month sales per share, higher than the industry average of 1.94 times.

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Movements in EPS estimates for 2026 and 2027 have been mixed over the past 30 days.

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BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Agreement Resolves all Pending Patent-Related Proceedings Between BioMarin and Ascendis

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has entered into binding terms with Ascendis Pharma A/S, resolving the patent and ancillary disputes pending globally, including before the U.S. International Trade Commission (ITC) concerning Ascendis's Yuviwel. As part of the agreement, Ascendis will pay BioMarin a royalty equal to 20% of net sales of Yuviwel in the U.S., retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil and South Korea until May 2030. 

"This outcome incentivizes companies like BioMarin to keep investing in the kind of long-term innovation that is critical to bringing breakthrough treatments to the people who need them," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We have spent decades focused on understanding the underlying biology of rare genetic conditions, building the deep scientific expertise that led to our development of six first-in-disease medicines for patients. We look forward to continuing to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on our ongoing momentum for children with achondroplasia."

The scope of the settlement includes a license for BioMarin's patents that relate to Yuviwel for all current and potential indications, including achondroplasia and hypochondroplasia. It also covers the use of Yuviwel in combination with other medicines. Under the terms of the agreement, BioMarin will dismiss the pending Section 337 investigation before the ITC and the parties will resolve all claims relating to the asserted intellectual property, including litigation pending in Brazil, Denmark, Germany, South Korea and the Northern District of California.

Reaching this agreement recognizes the value of BioMarin's pioneering innovations in C-type natriuretic peptide (CNP) technology, including the development of VOXZOGO® (vosoritide), while providing a framework that enables continued access to medicine for children with achondroplasia around the world.

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com. 

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the settlement and license agreement with Ascendis Pharma A/S, including expected benefits of such agreement and anticipated royalty payments, and future commercialization of licensed products and BioMarin's expectations to continue to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on its momentum for children with achondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's ability to enforce the agreement; actual sales of licensed products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Andrew Villani

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558 

(628) 269-7393

SOURCE BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc. (BMRN) Q2 2026 Earnings Call August 6, 2026 4:30 PM EDT

Company Participants

Traci McCarty - Group Vice President
Alexander Hardy - President, CEO & Director
Cristin Hubbard - Executive VP & Chief Commercial Officer
Gregory Friberg - Executive VP and Chief Research & Development Officer
Brian Mueller - CFO & Executive VP of Finance

Conference Call Participants

Christopher Raymond - Raymond James & Associates, Inc., Research Division
Cory Kasimov - Evercore ISI Institutional Equities, Research Division
Jessica Fye - JPMorgan Chase & Co, Research Division
Tommie Reerink - Goldman Sachs Group, Inc., Research Division
Philip Nadeau - TD Cowen, Research Division
Eliana Merle - Barclays Bank PLC, Research Division
Mohit Bansal - Wells Fargo Securities, LLC, Research Division
Phoebe Tan - Jefferies LLC, Research Division
Paul Matteis - Stifel, Nicolaus & Company, Incorporated, Research Division
Sean Laaman - Morgan Stanley, Research Division
Alexandria Hammond - Wolfe Research, LLC

Presentation

Operator

Good afternoon, and welcome, everyone, to the BioMarin Pharmaceutical Second Quarter 2026 Conference Call. Today's conference is being recorded. [Operator Instructions]

At this time, I would like to turn the conference over to Traci McCarty, Head of Investor Relations.

Traci McCarty
Group Vice President

Thank you, operator, and thank you all for joining us today. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports.

In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today. These non-GAAP

Second Quarter 2026 Total Revenues Increased 20% Year-over-year to $990 million

Stronger Growth Expectations Drive Increased Guidance for Full-year 2026 Total Revenues, VOXZOGO®, and Non-GAAP Diluted Earnings Per Share (EPS)

Addition of GALAFOLD® and POMBILITI® + OPFOLDA®, with Cost Synergies, Expected to Accelerate Revenue Growth, Non-GAAP Diluted EPS Accretion, Non-GAAP Operating Margin Expansion, and Operating Cash Flow through the Mid-2030s

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

, /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the second quarter ended June 30, 2026.

"This quarter, we executed strongly across our portfolio while rapidly integrating Amicus into BioMarin's operations and advancing plans to accelerate growth for GALAFOLD and POMBILITI + OPFOLDA, and extending the benefit of these medicines to more patients worldwide," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand led us to increase full-year VOXZOGO revenue guidance to at least $1 billion in 2026.  Adding to this momentum is the opportunity to advance our second potential indication with VOXZOGO, hypochondroplasia, based on recent pivotal data that exceeded our expectations." Mr. Hardy added, "With our larger, more diversified commercial portfolio of innovative medicines, we are positioned to deliver additional growth and increased profitability. We expect strong execution through the remainder of 2026, bringing together our expanded portfolio, scale and disciplined integration efforts to reach more patients living with serious genetic conditions around the world."

2026 Business and Pipeline Highlights

Innovation

BioMarin recently submitted its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia. If approved, VOXZOGO would be the first targeted therapy for the treatment of hypochondroplasia, with a potential 2027 launch. The company plans to provide an update on the application status as part of its third quarter earnings update. In May, the company announced that the Phase 3 CANOPY-HCH-3 study of VOXZOGO in children with hypochondroplasia met its primary endpoint, with a statistically significant increase in annualized growth velocity (AGV) at week 52 versus placebo (LS mean difference +2.33 cm/yr, p<0.0001), along with significant improvements in standing height, height Z-score, and the key secondary endpoint of arm span. The full Phase 3 dataset will be shared in a late-breaking oral presentation at the European Society for Paediatric Endocrinology Annual Meeting in September. In June, at the Endocrine Society Annual Meeting (ENDO 2026), a Phase 2 investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia showed sustained improvements in growth with a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through year three, with mean height standard deviation score (SDS) improving 0.72 over the three years. Also at ENDO 2026, the company presented Phase 1 data for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a single-ascending-dose study in healthy adults, BMN 333 demonstrated sustained exposure supporting weekly dosing and was well tolerated, with free CNP exposure at the highest dose more than 13-fold that of another long-acting CNP agent, reflecting its potential to become a new standard of care in achondroplasia. The Phase 2/3 study is enrolling, with a data update expected in 2027. In July, BioMarin announced that the FDA accepted its sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027. In the second quarter, the European Commission approved PALYNZIQ® for adolescents 12 years and older with phenylketonuria (PKU). PALYNZIQ is the only therapy that enables people with PKU to reach physiologic Phe levels while reducing dietary restrictions, regardless of severity. During the quarter, BioMarin added BMN 820 (formerly DMX-200) to its portfolio, a first-in-class oral CCR2 inhibitor for focal segmental glomerulosclerosis (FSGS) for which BioMarin holds exclusive U.S. commercialization rights. BMN 820 has the potential to treat a broad FSGS population, regardless of nephrotic syndrome status, and represents a U.S. total addressable patient population of approximately 30,000. The Phase 3 ACTION 3 trial is ongoing, with pivotal data expected in 2028. BMN 351, BioMarin's Phase 1/2 candidate for Duchenne muscular dystrophy, continued in development. The company expects to provide a program update by year-end. Following the pivotal ENERGY 3 trial results, previously announced in May, in which BMN 401 did not meet one of its two co-primary endpoints for the treatment of ENPP1 deficiency, BioMarin has now made the decision to discontinue development of BMN 401 across all indications. In July, BioMarin and the n-Lorem Foundation entered a collaboration and global exclusive license agreement to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. ReNU syndrome has an expected global population of approximately 100,000. Growth

BioMarin expects peak revenue for GALAFOLD to be approximately $1.4 billion by the mid-2030s and for POMBILITI + OPFOLDA to be approximately $1.2 billion by the mid-to-late-2030s. BioMarin expects these high growth therapies to benefit from its global scale and proven commercial capabilities. Metabolic Conditions (formerly Enzyme Therapies) revenue grew 25% Y/Y in the second quarter of 2026, driven by the additions of GALAFOLD and POMBILITI + OPFOLDA and continued strength from PALYNZIQ. The number of patients on therapy grew across all BioMarin-marketed therapies, both Y/Y and sequentially. Strong U.S. and global demand led to increased full-year 2026 VOXZOGO revenue guidance to a low end of $1 billion. The number of children being treated with VOXZOGO globally increased by more than 20% Y/Y in the second quarter. In the U.S., the majority of new patient starts were under two years of age, and the region drove approximately 25% of total VOXZOGO revenue during the quarter. Value Commitment

As part of the acquisition of Amicus, which closed on April 27, 2026, the company identified approximately $280 million of cost reductions on a GAAP basis, and approximately $220 million of cost reductions on a Non-GAAP basis, expected to be fully realized in 2028, representing an approximately 50% reduction from Amicus-reported 2025 GAAP and Non-GAAP operating expenses, respectively. Synergies reflect a reduction of Amicus' legacy labor costs and external spend and are expected to be largely driven by general and administrative functions, with the large majority of sales and marketing capabilities retained to support continued commercial growth. GALAFOLD and POMBILITI + OPFOLDA, combined, are expected to reach over 60% Non-GAAP Operating Margin by 2030. The company is targeting gross leverage below 2.5 times by mid-year 2027, an acceleration by approximately one year of prior timeline guidance provided at deal announcement, supported by profitability growth of the combined company. Second Quarter 2026 Financial Highlights

Total Revenues for the second quarter of 2026 were $990 million, an increase of $165 million compared to the same period in 2025, primarily driven by revenues from GALAFOLD and POMBILITI + OPFOLDA, which were acquired from Amicus on April 27, 2026, as well as new patients initiating VOXZOGO therapy across all regions and growth in U.S. patients treated with PALYNZIQ. These increases were partially offset by lower VIMIZIM® revenue due to the timing of large government orders outside the U.S. and lower ALDURAZYME® sales volume due to the timing of order fulfillment to Sanofi. GAAP Net Income for the second quarter of 2026 decreased to $45 million compared to $241 million for the same period in 2025. The decrease was primarily driven by the acquisition of Amicus, including integration and restructuring costs, intangible asset amortization, interest expense from debt issued to finance a portion of the transaction, and amortization of inventory fair value step-up. Other drivers included higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO and higher Research and Development (R&D) spend related to BMN 401, which was acquired in the third quarter of 2025, partially offset by higher gross profit driven by revenue growth as described above. Non-GAAP Income for the second quarter of 2026 decreased to $236 million compared to $282 million for the same period in 2025. The decrease was primarily driven by higher interest expense, higher sales and marketing spend to support newly acquired products and global expansion of VOXZOGO, and higher R&D spend related to BMN 401, partially offset by higher gross profit driven by revenue growth as described above. Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

% Change

2026

2025

% Change

Total Revenues

$990

$825

20 %

$1,756

$1,571

12 %

Net Product Revenues by Product:

VOXZOGO

$253

$221

14 %

$472

$435

9 %

Metabolic Conditions:

VIMIZIM

$194

$215

(10) %

$405

$404

— %

NAGLAZYME®

135

129

5 %

265

243

9 %

PALYNZIQ

135

106

27 %

225

199

13 %

GALAFOLD

106

—

NM

106

—

NM

BRINEURA®

51

49

4 %

98

89

10 %

ALDURAZYME

44

56

(21) %

80

105

(24) %

POMBILITI + OPFOLDA

30

—

NM

30

—

NM

Total Metabolic Conditions Revenue

$695

$555

25 %

$1,209

$1,040

16 %

KUVAN®

$24

$27

(11) %

$48

$52

(8) %

ROCTAVIAN®(1)

$12

$9

33 %

$14

$20

(30) %

GAAP Net Income

$45

$241

(81) %

$150

$426

(65) %

Non-GAAP Income (2)

$236

$282

(16) %

$385

$502

(23) %

GAAP Operating Margin % (3)

11.2 %

33.5 %

13.7 %

31.9 %

Non-GAAP Operating Margin % (2)

36.4 %

39.9 %

31.0 %

37.9 %

GAAP Diluted EPS

$0.23

$1.23

(81) %

$0.77

$2.19

(65) %

Non-GAAP Diluted EPS (2)

$1.20

$1.44

(17) %

$1.96

$2.57

(24) %

NM

Percentage change is not meaningful for products acquired from Amicus on April 27, 2026.

(1)

In 2026, the company announced that it will no longer market ROCTAVIAN.

(2)

Refer to Non-GAAP Information beginning on page 10 of this press release for definitions of Non-GAAP Income, Non-GAAP Operating Margin percentage and Non-GAAP Diluted EPS along with the related reconciliations to the comparable information reported under U.S. GAAP.

(3)

GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues.

Forward-Looking Non-GAAP Financial Information

BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from its strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.

Updated 2026 Full-Year Financial Guidance (in millions, except EPS amounts)

Total Revenues, VOXZOGO, and Non-GAAP Diluted EPS guidance raised, reflecting strong first-half 2026 performance and second-half 2026 revenue expectations for both Metabolic Conditions and VOXZOGO. Guidance reflects post-close contributions from Amicus beginning April 27, 2026. BioMarin will continue to include interest expense related to the Amicus financing in both GAAP and Non‑GAAP financial results. Based on current rates, interest expense associated with the financing is estimated at approximately $200 million on an annualized basis, with Term Loans and Senior Notes scheduled to mature after 2030. Item

Provided on May 4, 2026

Updated August 6, 2026

Midpoint Growth
(Y/Y)

Total Revenues

$3,825

to

$3,925

$3,875

to

$3,925

21 %

Metabolic Conditions

$2,725

to

$2,775

Unchanged

31 %

VOXZOGO

$975

to

$1,025

$1,000

to

$1,050

11 %

Other Revenues(1)

$100

to

$125

Unchanged

Non-GAAP Diluted EPS (2)(3)(4)

$4.85

to

$5.05

$4.90

to

$5.10

59 %

(1)

Other Revenues includes KUVAN, ROCTAVIAN, and royalties.

(2)

Refer to Non-GAAP Information beginning on page 10 of this press release for definition of Non-GAAP Diluted EPS.

(3)

Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.

(4)

Non-GAAP Diluted EPS guidance assumes a combined company tax rate of 22%.

BioMarin will host a conference call and webcast to discuss second quarter 2026 financial results today, Thursday, August 6, 2026, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

U.S./Canada Dial-in Number: 800-715-9871

Replay Dial-in Number: 800-770-2030

International Dial-in Number:  646-307-1963

Replay International Dial-in Number: 609-800-9909

Conference ID:  3551298 

Conference ID: 3551298 

About BioMarin

BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit www.biomarin.com.

Forward-Looking Statements

This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: future financial performance, including the expectations of Total Revenues, Non-GAAP Diluted EPS, Non-GAAP Operating Margin, gross leverage, operating cash flow and revenue compound annual growth rate (CAGR) for, in certain instances, the full-year 2026, fourth quarter and second half of 2026, and future periods, and the underlying drivers of those results, such as the expected demand and continued growth of BioMarin's Metabolic Conditions portfolio, including PALYNZIQ, and VOXZOGO, and the expected impact of the acquisition of Amicus Therapeutics, Inc. (Amicus); the anticipated benefits of the acquisition of Amicus, including the expected amount and timing of cost synergies as well as expected revenue from the addition of GALAFOLD and POMBILITI + OPFOLDA, including BioMarin's plans and expectations to accelerate growth through mid-2030s; BioMarin's plans for investment in innovation and future growth; the timing of orders for commercial products; plans and expectations regarding the development, commercialization and commercial prospects of BioMarin's product candidates and commercial products, including the prospects and timing of actions relating to clinical studies and trials and product approvals, such as study initiations, study advancements, data readouts, submissions, filings, approvals, and label expansions; the expected benefits and availability of BioMarin's commercial products and product candidates, including with respect to the potential new indication for VOXZOGO in hypochondroplasia; and potential growth opportunities and trends, including the assumptions and expectations regarding total addressable patient population (TAPP) with respect to the conditions targeted by BioMarin's product candidates and commercial products.

These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products; BioMarin's ability to realize the anticipated benefits of any acquisitions; BioMarin's ability to accurately estimate future financial performance; impacts of macroeconomic and other external factors on BioMarin's operations, regulatory uncertainty, the impact of new or increased tariffs, other trade protection measures, and escalating trade tensions; geopolitical instability, wars and military conflicts; results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities concerning each of the described products and product candidates; the market for each of these products; BioMarin's ability to meet product demand; actual sales of BioMarin's commercial products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin®, VOXZOGO®, VIMIZIM®, NAGLAZYME®, PALYNZIQ®, BRINEURA®, KUVAN®, ROCTAVIAN®, GALAFOLD®, and POMBILITI® + OPFOLDA® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF INCOME

Three and Six Months Ended June 30, 2026 and 2025

(In thousands of U.S. dollars, except per share amounts)

(Unaudited)

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

REVENUES:

Net product revenues

$         984,393

$         812,982

$       1,744,471

$       1,547,626

Royalty and other revenues

5,315

12,428

11,445

22,929

Total revenues

989,708

825,410

1,755,916

1,570,555

OPERATING EXPENSES:

Cost of sales

202,795

150,090

397,794

301,648

Research and development

206,952

161,308

385,748

320,039

Selling, general and administrative

395,549

232,279

653,839

438,395

Intangible asset amortization

73,492

4,846

77,975

9,693

Total operating expenses

878,788

548,523

1,515,356

1,069,775

INCOME FROM OPERATIONS

110,920

276,887

240,560

500,780

Interest income

10,480

18,827

33,040

37,840

Interest expense

(63,295)

(2,679)

(78,253)

(5,542)

Other income, net

3,279

4,833

7,240

2,879

INCOME BEFORE INCOME TAXES

61,384

297,868

202,587

535,957

Provision for income taxes

16,622

57,336

52,298

109,739

NET INCOME

$          44,762

$        240,532

$        150,289

$        426,218

EARNINGS PER SHARE, BASIC

$              0.23

$              1.25

$              0.78

$              2.23

EARNINGS PER SHARE, DILUTED

$              0.23

$              1.23

$              0.77

$              2.19

Weighted average common shares outstanding, basic

193,423

191,907

192,959

191,440

Weighted average common shares outstanding, diluted

194,467

197,091

194,147

196,643

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

June 30, 2026 and December 31, 2025

(In thousands of U.S. dollars, except per share amounts)

(Unaudited)

June 30, 2026

December 31, 2025

ASSETS

Current assets:

Cash and cash equivalents

$              874,005

$            1,311,679

Short-term investments

—

248,930

Accounts receivable, net

1,061,047

908,214

Inventory

1,782,524

1,298,883

Other current assets

254,047

185,784

Total current assets

3,971,623

3,953,490

Noncurrent assets:

Long-term investments

—

492,242

Property, plant and equipment, net

989,994

952,508

Intangible assets, net

4,879,367

213,837

Goodwill

655,745

196,199

Deferred tax assets

888,575

1,508,697

Other assets

338,138

277,049

Total assets

$           11,723,442

$            7,594,022

LIABILITIES AND STOCKHOLDERS' EQUITY

Current liabilities:

Accounts payable and accrued liabilities

$            1,005,793

$              759,031

Current portion of long-term debt, net

658,203

—

Total current liabilities

1,663,996

759,031

Noncurrent liabilities:

Long-term debt, net

3,527,939

597,176

Other long-term liabilities

209,815

150,816

Total liabilities

5,401,750

1,507,023

Stockholders' equity:

Common stock, $0.001 par value: 500,000,000 shares authorized; 193,535,556 and 192,300,101 shares issued and outstanding, respectively

194

192

Additional paid-in capital

6,037,019

5,956,582

Company common stock held by the Nonqualified Deferred Compensation Plan

(11,233)

(10,508)

Accumulated other comprehensive income (loss)

(8,783)

(13,473)

Retained earnings

304,495

154,206

Total stockholders' equity

6,321,692

6,086,999

Total liabilities and stockholders' equity

$           11,723,442

$            7,594,022

BIOMARIN PHARMACEUTICAL INC.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Six Months Ended June 30, 2026 and 2025

(In thousands of U.S. dollars)

(Unaudited)

Six Months Ended June 30,

2026

2025

CASH FLOWS FROM OPERATING ACTIVITIES:

Net income

$          150,289

$          426,218

Adjustments to reconcile net income to net cash provided by operating activities:

Depreciation and amortization

114,049

40,632

Non-cash interest expense

27,912

1,320

Stock-based compensation

119,152

85,231

Impairment of assets

—

2,967

Deferred income taxes

2,261

61,771

Unrealized foreign exchange gains

(4,046)

(5,306)

Other

(5,534)

(4,633)

Changes in operating assets and liabilities, net of effects of business acquired:

Accounts receivable, net

(46,005)

(156,124)

Inventory

12,334

(72,462)

Other current assets

(23,589)

(15,092)

Other assets

9,005

(13,505)

Accounts payable and accrued liabilities

26,265

3,111

Other long-term liabilities

6,667

5,537

Net cash provided by operating activities

388,760

359,665

CASH FLOWS FROM INVESTING ACTIVITIES:

Purchases of property, plant and equipment

(49,739)

(33,869)

Maturities and sales of investments

767,277

195,738

Purchases of investments

(25,792)

(202,433)

Purchase of intangible assets

(5,433)

(266)

Acquisition of Amicus, net of cash acquired

(5,067,630)

—

Other

4,966

—

Net cash used in investing activities

(4,376,351)

(40,830)

CASH FLOWS FROM FINANCING ACTIVITIES:

Proceeds from exercises of awards under equity incentive plans

6,655

7,707

Taxes paid related to net share settlement of equity awards

(39,504)

(51,089)

Proceeds from borrowings

3,650,000

—

Payments of debt issuance costs

(65,604)

—

Net cash provided by (used in) financing activities

3,551,547

(43,382)

Effect of exchange rate changes on cash

(1,630)

(4,479)

NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS

(437,674)

270,974

Cash and cash equivalents:

Beginning of period

$        1,311,679

$          942,842

End of period

$          874,005

$        1,213,816

Non-GAAP Information
The results presented in this press release include both GAAP information and Non-GAAP information. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization, stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Cost of Sales (COS), Non-GAAP R&D expenses and Non-GAAP Selling, General and Administrative (SG&A) expenses are defined by the company as GAAP COS, GAAP R&D expenses and GAAP SG&A expenses, respectively, excluding stock-based compensation expense and, in certain periods, certain other specified items, as detailed below when applicable. Non-GAAP Operating Margin percentage is defined by the company as GAAP Income (Loss) from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP Weighted-Average Diluted Shares Outstanding. Non-GAAP Weighted-Average Diluted Shares Outstanding is defined by the company as GAAP Weighted-Average Diluted Shares Outstanding, adjusted to include any common shares issuable under the company's equity plans or convertible debt in periods when they are dilutive under Non-GAAP. Projected Gross Leverage is defined by the company as undiscounted debt (total debt excluding unamortized discount and deferred offering costs) as of the balance sheet date divided by 4 quarter projected Non-GAAP Adjusted EBITDA. Non-GAAP Adjusted EBITDA is defined by the company as GAAP Income (or Loss) from Operations excluding the impact of depreciation, amortization and stock-based compensation expense. Non-GAAP synergies is defined by the company as Amicus' legacy labor and external spend cost reductions, excluding the impact of stock-based compensation.

BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Because these Non-GAAP metrics are important internal measurements for BioMarin, the company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the company's results from period to period and to its forward-looking guidance, and to identify operating trends in the company's principal business.

Non-GAAP financial measures are not meant to be considered in isolation or as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the company may exclude for purposes of its Non-GAAP financial measures; likewise, the company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:

Reconciliation of GAAP Reported Information to Non-GAAP Information (1)

(In millions of U.S. dollars, except per share data)

(unaudited)

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

GAAP Reported Net Income

$         45

$        241

$        150

$        426

Adjustments

Stock-based compensation expense - COS

5

4

9

6

Stock-based compensation expense - R&D

16

14

28

26

Stock-based compensation expense - SG&A(2)

55

30

83

53

Amortization of intangible assets

73

5

78

10

Amortization of acquired inventory step-up(3)

12

—

12

—

Acquisition-related costs(3)

84

—

84

—

Severance costs (4)

3

—

12

—

Loss on investments (5)

—

—

—

3

Income tax effect of adjustments

(57)

(11)

(70)

(22)

Non-GAAP Income

$        236

$        282

$        385

$        502

Three Months Ended

June 30,

2026

2025

COS

R&D

SG&A

COS

R&D

SG&A

GAAP expenses

$     203

$      207

$      396

$      150

$      161

$       232

Adjustments

Stock-based compensation expense(2)

(5)

(16)

(55)

(4)

(14)

(30)

Amortization of acquired inventory step-up(3)

(12)

—

—

—

—

—

Acquisition-related costs (3)

—

—

(84)

—

—

—

Severance costs (4)

—

—

(3)

—

—

—

Non-GAAP expenses

$     186

$      191

$      253

$      146

$      147

$       203

Six Months Ended

June 30,

2026

2025

COS

R&D

SG&A

COS

R&D

SG&A

GAAP expenses

$      398

$      386

$      654

$      302

$      320

$      438

Adjustments

Stock-based compensation expense(2)

(9)

(28)

(83)

(6)

(26)

(53)

Amortization of acquired inventory step-up(3)

(12)

—

—

—

—

—

Acquisition-related costs (3)

—

—

(84)

—

—

—

Severance costs (4)

—

—

(12)

—

—

—

Non-GAAP expenses

$      378

$      358

$      475

$      295

$      294

$      385

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

Percent
of GAAP
Total
Revenue

2025

Percent
of GAAP
Total
Revenue

2026

Percent
of GAAP
Total
Revenue

2025

Percent
of GAAP
Total
Revenue

GAAP Income from Operations

$      111

11.2 %

$     277

33.5 %

$     241

13.7 %

$     501

31.9 %

Adjustments

Stock-based compensation expense(2)

76

7.7

48

5.8

120

6.8

85

5.4

Amortization of intangible assets

73

7.4

5

0.6

78

4.4

10

0.6

Amortization of acquired inventory step-up(3)

12

1.2

—

—

12

0.7

—

—

Acquisition-related costs (3)

84

8.5

—

—

84

4.8

—

—

Severance costs (4)

3

0.3

—

—

12

0.7

—

—

Non-GAAP Income from Operations

$      360

36.4 %

$     329

39.9 %

$     545

31.0 %

$     596

37.9 %

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

GAAP Diluted EPS

$       0.23

$       1.23

$       0.77

$       2.19

Adjustments

Stock-based compensation expense(2)

$       0.38

$       0.24

0.60

0.43

Amortization of intangible assets

$       0.37

$       0.03

0.39

0.05

Amortization of acquired inventory step-up(3)

$       0.06

$          —

0.06

—

Acquisition-related costs(3)

$       0.42

$          —

0.42

—

Severance costs (4)

$       0.02

$          —

0.06

—

Loss on investments (5)

$          —

$          —

—

0.02

Income tax effect of adjustments

$      (0.29)

$      (0.06)

(0.35)

(0.11)

Non-GAAP Diluted EPS

$       1.20

$       1.44

$       1.96

$       2.57

Three Months Ended

June 30,

Six Months Ended

June 30,

2026

2025

2026

2025

GAAP Weighted-Average Diluted Shares Outstanding

194.5

197.1

194.1

196.6

Adjustments

Common stock issuable under company's convertible debt (6)

4.4

—

4.4

—

Non-GAAP Weighted-Average Diluted Shares Outstanding

198.9

197.1

198.5

196.6

(1)

Certain amounts may not sum or recalculate due to rounding.

(2)

Stock-based compensation expense recorded in SG&A for the three and six months ended June 30, 2026, includes approximately $13 million related to the post-combination service period for unvested Amicus stock options.

(3)

These amounts represent costs resulting from the Amicus acquisition that closed on April 27, 2026. Acquisition-related costs were included in SG&A and consisted of severance, transaction and integration costs. Amortization of acquired inventory step-up was included in COS.

(4)

These amounts were included in SG&A and represent charges for severance in connection with the company's plan to simplify its organizational design and strategic initiatives in the first and second quarters of 2026.

(5)

Represents impairment loss on non-marketable equity securities recorded in Other income, net, in the first quarter of 2025.

(6)

Common stock issuable under the company's convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three and six months ended June 30, 2026 as they were anti-dilutive.

Amicus Therapeutics, Inc. (1)

Reconciliation of Non-GAAP Financial Measures

(in thousands)

(Unaudited)

Twelve Months Ended

December 31, 2025

Total operating expenses - as reported GAAP

$                528,492

Research and development:

Share-based compensation

12,156

Selling, general and administrative:

Share-based compensation

75,254

Loss on impairment of assets

1,702

Depreciation and amortization

7,460

Total operating expense adjustments to reported GAAP

96,572

Total operating expenses - as adjusted

$                431,920

(1)

The above historical reconciliation is reproduced from Amicus' earnings release furnished as Exhibit 99.1 to its Current Report on Form 8-K dated February 20, 2026 and reflects Amicus' historical definitions of the applicable non-GAAP measures.

Contact:

Investors:

Media:

Traci McCarty

Marni Kottle

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(650) 374-2803

SOURCE BioMarin Pharmaceutical Inc.

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.

Contacts:

Investors             

Media

Traci McCarty              

Andrew Villani

BioMarin Pharmaceutical Inc.            

BioMarin Pharmaceutical Inc.

(415) 455-7558                                     

(628) 269-7393

SOURCE BioMarin Pharmaceutical Inc.