BioMarin Pharmaceutical Inc (BMRN)

BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with HypochondroplasiaMay 20, 2026 4:20 PM
PR Newswire (US) 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 independenceSafety findings consistent with the established profile in achondroplasia with no new safety signals identifiedSupplemental 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 SAN RAFAEL, Calif., May 20, 2026 /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

BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 DeficiencyMay 18, 2026 9:05 AM
PR Newswire (US) 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. Deficiency in ENPP1 function results in a decrease in plasma inorganic pyrophosphate (PPi) that can lead to progressive damage to blood vessels, soft tissues and bones. The study met one co-primary endpoint, showing treatment with BMN 401 resulted in statistically significant increases in plasma PPi through week 52 compared to the conventional therapy control arm in children with ENPP1 deficiency. However, the study did not meet its other co-primary endpoint as there was no corresponding improvement in Radiographic Global Impression of Change (RGI-C) scores, an important measure of treatment impact in children with rickets. Additionally, no positive trends were observed across secondary endpoints, including Rickets Severity Score (RSS) and growth Z-score (height/body length and weight). BMN 401 was generally well-tolerated with no new safety signals reported."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, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "We are actively evaluating these data to determine the appropriate next steps. ENPP1 deficiency is a devastating disease, particularly for infants, where mortality rates remain high and new treatment options are urgently needed. We are deeply grateful to the children, families, investigators and study teams who are participating in this trial."Detailed results from the ENERGY 3 study will be presented at an upcoming medical meeting.About BMN 401 and ENERGY 3BMN 401 (formerly INZ-701) is a potential first-in-class, subcutaneous enzyme replacement therapy for the treatment of people with ENPP1 deficiency.ENERGY 3 is a Phase 3 multicenter randomized (2:1) controlled open-label clinical trial designed to evaluate the efficacy and safety of BMN 401 in children ages 1-12 with ENPP1 deficiency. Change from baseline in PPi through week 52 and RGI-C global score at week 52 are co-primary endpoints in this study. Following discussions with health authorities, RGI-C was added as a co-primary endpoint to assess clinically meaningful functional improvement in children with ENPP1 deficiency.Key secondary endpoints include change from baseline in Rickets Severity Score (RSS), change from baseline in growth Z-score (height/body length and weight), and measurements of BMN 401 serum concentration and enzyme activity. Enrollment in ENERGY 3 was completed in January 2025, with the inclusion of 27 pediatric participants.For more information, please visit clinicaltrials.biomarin.com.About ENPP1 DeficiencyENPP1 deficiency is a rare and lifelong genetic condition caused by changes in the ENPP1 gene, leading to progressive damage to blood vessels, soft tissues and bones. Infants with this condition are often diagnosed with generalized arterial calcification of infancy (GACI) Type 1, and about 50% of them do not survive beyond six months. Children with ENPP1 deficiency typically develop a type of rickets called autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia (softened bones), both of which may cause pain and difficulty with movement. Individuals may also experience hearing loss, calcification in arteries and joints, and heart problems.About BioMarinBioMarin 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 StatementsThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin's plans and expectations regarding the BMN 401 program generally, and the pivotal Phase 3 ENERGY 3 trial results particularly; the safety profile and potential benefits of BMN 401; and BioMarin's plans to share detailed results from the ENERGY 3 study at an upcoming medical meeting. 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.Contacts:


Investors                                                                             MediaTraci McCarty                                                                       Andrew VillaniBioMarin Pharmaceutical Inc.                                               BioMarin Pharmaceutical Inc.(415) 455-7558                                                                   (628) 269-7393 View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-provides-update-on-phase-3-trial-for-bmn-401-in-children-aged-1-12-with-enpp1-deficiency-302774207.htmlSOURCE BioMarin Pharmaceutical Inc. Original: BioMarin Provides Update on Phase 3 Trial for BMN 401 in Children Aged 1-12 With ENPP1 Deficiency

BioMarin Reports First Quarter 2026 Financial and Operating ResultsMay 4, 2026 4:05 PM
PR Newswire (US) First Quarter 2026 Total Revenues Increased Year-over-year to $766 millionIncreased 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 PortfolioConference Call and Webcast Scheduled Today at 4:30 p.m. ETSAN RAFAEL, Calif., May 4, 2026 /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 HighlightsInnovationIn 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

BioMarin Presents New Data on the Effect of Long-Term Treatment with VOXZOGO® (vosoritide) on Arm Span, Bone Health and Growth in Children With Achondroplasia at the Pediatric Endocrine Society's 2026 Annual MeetingMay 2, 2026 3:30 PM
PR Newswire (US)

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 achondroplasiaResearchers will present additional data from studies of VOXZOGO for hypochondroplasia, ahead of pivotal topline Phase 3 data expected in the first half of 2026 SAN RAFAEL, Calif., May 2, 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 MeasuresData 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

BioMarin Completes Acquisition of Amicus TherapeuticsApril 27, 2026 8:44 AM
PR Newswire (US)

Acquisition Adds Galafold® (migalastat) for Fabry Disease and Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) for Pompe Disease to BioMarin's Commercial PortfolioBioMarin Expects to Provide Updated FY 2026 Guidance During its First Quarter Earnings Call, May 4, 2026SAN RAFAEL, Calif., April 27, 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 INFORMATIONADVERSE 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) 455-7558Media
Marni Kottle
BioMarin Pharmaceutical Inc.
(415) 218-7111




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Original: BioMarin Completes Acquisition of Amicus Therapeutics

BioMarin to Host First Quarter 2026 Financial Results Conference Call and Webcast on Monday, May 4, 2026, at 4:30pm ETApril 27, 2026 7:30 AM
PR Newswire (US)

SAN RAFAEL, Calif., April 27, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President and Chief Executive Officer of BioMarin, will host a conference call and webcast on Monday, May 4, 2026, at 4:30 p.m. ET to discuss first quarter 2026 financial results and provide a general business update.







Dial-in Number
U.S. / Canada Dial-in Number: 800-715-9871
International Dial-in Number: 646-307-1963
Conference Call ID: 3424435 U.S. / Canada Replay Dial-in Number: 800-770-2030
International Replay Dial-in Number: 609-800-9909
Playback ID: 3424435  Interested parties may access a live audio webcast of the conference call via the investor section of the BioMarin website, https://investors.biomarin.com/. A replay of the call will be archived on the site for one week following the call.About BioMarin
BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with 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.  Contacts:




InvestorsMedia
Traci McCartyMarni Kottle
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 455-7558(650) 374-2803
 



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Original: BioMarin to Host First Quarter 2026 Financial Results Conference Call and Webcast on Monday, May 4, 2026, at 4:30pm ET

BioMarin Announces Board Leadership TransitionApril 21, 2026 4:23 PM
PR Newswire (US)

Ian T. Clark elected Chair of the Board; Richard A. Meier to retire SAN RAFAEL, Calif., April 21, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) said today that the Board of Directors has appointed Ian T. Clark as Chair of the Board, subject to his election at the company's Annual Meeting of Stockholders on June 2, 2026. Mr. Clark will succeed Richard A. Meier, who is retiring after two decades on the Board, and the appointment will become effective immediately following the Annual Meeting.







Mr. Clark joined BioMarin's Board in 2025 and brings extensive board, governance and executive leadership experience across the global biopharmaceutical industry. He has served on the boards of a number of public biopharmaceutical companies, including roles as lead independent director and chair. He was Chief Executive Officer of Genentech and a member of Genentech's Board of Directors from 2010 to 2016."I am excited to step into this role as BioMarin enters its next phase of growth as a leading rare disease company operating at scale in 80 countries," said Mr. Clark. "With two recent acquisitions, BioMarin has the potential to unlock significant value for all stakeholders: employees, shareholders and, most importantly, patients." Mr. Meier joined BioMarin's Board in 2006, playing a key role in the company's evolution. He was Lead Independent Director before being elected Chair in 2023. During this period, profitability improved meaningfully, with significant expansion in non-GAAP operating margin, earnings-per-share growth and substantial cash flow generation."BioMarin enters this next chapter with excellent leadership, strategic clarity and a strong foundation for continued success," said Mr. Meier. "The company is well positioned to build on this momentum in the years ahead. It has been an honor to serve on BioMarin's Board over the past two decades and I am pleased to hand the reins to Ian."Alexander Hardy, President and Chief Executive Officer of BioMarin, said, "Randy has helped guide BioMarin through a period of meaningful growth and transformation, and we are grateful for his leadership and service. I am pleased to welcome Ian to his new role. Ian brings substantial board and executive experience, and I look forward to partnering with him as we continue executing our strategy for patients 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 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 election of Ian T. Clark as Chair of the Board of Directors of BioMarin effective as of the Annual Meeting of Stockholders to be held on June 2, 2026, and subject to Mr. Clark's election at the Annual Meeting; and the future business prospects of BioMarin, including BioMarin's ability to unlock significant value for all stakeholders. 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 consummate and realize the anticipated benefits of any acquisitions; 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; 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 (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® is a registered trademark of BioMarin Pharmaceutical Inc.Contacts:


InvestorsMedia  Traci McCarty Marni KottleBioMarin Pharmaceutical Inc.                        BioMarin Pharmaceutical Inc. (415) 455-7558   (415) 218-7111



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Original: BioMarin Announces Board Leadership Transition

BioMarin Presents New Data on the Positive Impact of Early Treatment With VOXZOGO® (vosoritide) on Proportionality and Arm Span in Children with Achondroplasia at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics MMarch 12, 2026 9:05 AM
PR Newswire (US)

New results demonstrate early VOXZOGO treatment leads to durable and sustained improvements in skeletal growth-related health outcomes, including proportionality, arm span and body mass index, building on over 10,000 patient-years of data  VOXZOGO is the only approved treatment for children with achondroplasia starting at birth and has the most extensive published clinical evidence, including in measures beyond growth, among all achondroplasia treatmentsSAN RAFAEL, Calif., March 12, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced positive new data from studies of VOXZOGO® (vosoritide) in children with achondroplasia in ongoing clinical trials and real-world studies. The latest findings reinforce the importance of early treatment and the well-known, established efficacy and safety of VOXZOGO. The data will be presented at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Baltimore.







Early Treatment, Maximum Impact in Children Who Start Treatment Before Age 2New data will be presented from a study of children who started VOXZOGO before age 2, which showed that treatment was associated with durable, multi-year growth improvements, including body proportionality and arm span compared to untreated children, as well as improvements in height over time.For proportionality, measured by change from baseline in upper-to-lower body segment ratio (ULBR), the least squares mean difference versus untreated children showed improvement by year one in children initiating VOXZOGO under age 2, and this difference continued to improve each year through year four. Specifically, the least squares mean difference versus untreated children in year one was -0.33 (p=0.0005) in children initiating VOXZOGO under age 2, and -0.10 (p=0.042) in those initiating treatment with VOXZOGO under 6 months; by year four, this trend continued, with a least squares mean difference of -0.53 (p

U.S. Food and Drug Administration Approves BioMarin's PALYNZIQ® (pegvaliase-pqpz) for Adolescents 12 Years of Age and Older with Phenylketonuria (PKU)February 27, 2026 8:00 PM
PR Newswire (US)

Treatment with PALYNZIQ led to statistically significant blood phenylalanine (Phe) lowering compared to diet alone in pivotal Phase 3 PEGASUS studyPALYNZIQ is the only enzyme substitution therapy approved for the treatment of people with PKUSAN RAFAEL, Calif., Feb. 27, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics License Application (sBLA) for PALYNZIQ® (pegvaliase-pqpz) to include pediatric patients 12 years of age and older with phenylketonuria (PKU). PALYNZIQ is the only enzyme substitution therapy approved to reduce blood phenylalanine (Phe) concentrations in people with PKU.







"Adolescence is a period of increasing independence and academic demands, and represents a particularly challenging time for individuals with PKU. The ultra-restrictive diet required for PKU management may become unsustainable, and poor blood Phe control leads to adverse neurocognitive outcomes. PALYNZIQ is the only genotype-independent medication which may bring Phe into the normal range while allowing an unrestricted diet," said Stephanie Sacharow, M.D., Director, Dr. Harvey Levy Program for PKU and Related Conditions, Boston Children's Hospital. "In my clinic we have found that PALYNZIQ treatment adherence is even more successful in teens under age 18, while they are living at home with family support, and this approval allows us to extend this therapeutic option to adolescents who may benefit most." The FDA approval is based on data from PEGASUS, a Phase 3 multi-center open-label randomized controlled study evaluating the safety and efficacy of PALYNZIQ compared to diet alone in adolescents aged 12 to

BioMarin Reports Fourth Quarter and Full-year 2025 Financial and Operating ResultsFebruary 23, 2026 4:05 PM
PR Newswire (US)

Full-year 2025 Total Revenues Increased 13% Y/Y to $3.2 Billion, Led by 9% Revenue Growth for Enzyme Therapies and 26% Revenue Growth for VOXZOGO®Fourth Quarter 2025 Total Revenues Increased 17% Y/Y Led by 13% Revenue Growth for Enzyme Therapies and 31% Revenue Growth for VOXZOGOAnnounced Definitive Agreement to Acquire Amicus Therapeutics, including Galafold® for Fabry Disease and Pombiliti® + Opfolda® for Pompe Disease; Expected to Significantly Accelerate and Diversify RevenuesBioMarin Provides 2026 Guidance Excluding any Post-Close Contribution from the Announced Acquisition of Amicus, Anticipated to Close in Q2'26Conference Call and Webcast Scheduled Today at 4:30 p.m. ETSAN RAFAEL, Calif., Feb. 23, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the fourth quarter and full year ended December 31, 2025.







"In 2025, operational excellence led to strengthening financial results, including double-digit topline growth, strong profitability and increasing cash flow. We also advanced multiple medicines in our pipeline and closed the year by announcing the acquisition of Amicus," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "The Amicus transaction, which is expected to close in the second quarter, represents a compelling opportunity to reach more patients around the world and further strengthen our revenue growth through the next decade.""We expect to build on this success in 2026, with another year of strong financial performance and momentum across the business. We look forward to adding Galafold and Pombiliti + Opfolda to our growing commercial enzyme therapies business, and to continued strong growth from VOXZOGO. Beyond our current commercial portfolio, we are excited by the progress we are seeing across our R&D pipeline and look forward to a multitude of pipeline catalysts throughout the year. These include three major data read-outs to support regulatory approvals, two age label expansions, plus the advancement of multiple clinical programs position us for significant portfolio progress. We are energized by what lies ahead this year and intend to deliver again on an ambitious set of priorities, demonstrating our dedication to innovation and sustained growth in ways that we believe will benefit patients, employees, and shareholders."2025 Business HighlightsInnovationAccelerated development of BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP), with Phase 1 PK data exceeding targeted free CNP exposure levels, reflecting its potential to become the new standard of care in achondroplasia.Advanced five new VOXZOGO indications within the CANOPY program, including a pivotal Phase 3 study in hypochondroplasia and Phase 2 studies in idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.Reported positive data from the PALYNZIQ® Phase 3 PEGASUS study in 12- to 17-year-olds demonstrating statistically significant reductions in blood phenylalanine (Phe) compared to diet alone for adolescents with PKU.Progressed BMN 351 for Duchenne muscular dystrophy, with initial Phase 1/2 data demonstrating 5.0% mean absolute dystrophin expression (without double-correction for histologic adjustment for muscle content) at week 25 in the 9 mg/kg cohort. The 12 mg/kg dose cohort continues to enroll participants, with topline data readout from this cohort expected in 2H'26.GrowthStrong patient demand across the portfolio fueled 13% Y/Y full-year 2025 total revenue growth.Enzyme Therapies full-year 2025 revenue advanced 9% Y/Y, supported by sustained high market penetration and patient adherence, led by robust 22% Y/Y growth from PALYNZIQ.VOXZOGO generated 26% Y/Y revenue growth for full-year 2025, driven by deeper market penetration and increasing demand for the treatment of achondroplasia across 55 commercial markets. Markets outside of the U.S. (OUS) drove approximately 73% of VOXZOGO revenue in full-year 2025, reflecting the therapy's strong uptake across global markets.Value CommitmentAnnounced the acquisition of Amicus Therapeutics in December 2025, expected to close in Q2'26, subject to regulatory clearances, approval by the stockholders of Amicus and other customary closing conditions. The addition of high-growth products, Galafold for Fabry Disease and Pombiliti + Opfolda for Pompe Disease, is expected to accelerate BioMarin's revenue growth and increase profitability. Adolescent label expansion for Pombiliti + Opfolda is anticipated in 2H'26.Generated operating cash flows totaling $100 million in fourth quarter 2025 and $828 million for the full year. Total cash and investments totaled approximately $2 billion at year-end, and continued increasing operating cash flow is expected to support sustained investment in innovation and future growth.Strong performance in 2025 led to significant GAAP and Non-GAAP Diluted Earnings per Share expansion, excluding acquired in-process research and development (IPR&D) charges related to the acquisition of Inozyme, $1.10 per share, and an inventory write-off related to ROCTAVIAN®, totaling $0.46 per share after tax.The company secured financing of approximately $3.7 billion of non-convertible debt to support the Amicus acquisition with strong demand, achieving favorable pricing across the capital structure.Anticipated 2026 Program UpdatesVOXZOGO:Phase 3 hypochondroplasia data 1H'26; regulatory submissions 2H'26U.S. supplemental new drug application (sNDA) for full approval of VOXZOGO in achondroplasia Q2'26Advancing Phase 2 studies in idiopathic short stature, Noonan syndrome, Turner syndrome, SHOX deficiencyBMN 333 (long-acting CNP):Initiate registration-enabling Phase 2/3 study in achondroplasia 1H'26PALYNZIQ:U.S. PDUFA date for the adolescent label expansion February 28, 2026; EU approval 2026BMN 401:Phase 3 topline data in 1 to 12 year-old population with ENPP1 deficiency 1H'26; global regulatory submissions 2H'26; potential first-in-disease launch 2027BMN 351Phase 1/2 data presentation for 6 mg/kg and 9 mg/kg cohorts at Muscular Dystrophy Association (MDA) Clinical & Scientific Congress (March 8–11, 2026)ROCTAVIANFollowing the company's October announcement to explore options to divest ROCTAVIAN, BioMarin undertook a comprehensive effort to identify a potential buyer. Despite these efforts, BioMarin was unable to identify a qualified buyer and has made the decision to voluntarily withdraw ROCTAVIAN from the market.Fourth Quarter 2025 Financial HighlightsTotal Revenues for the fourth quarter of 2025 were $875 million, an increase of 17% compared to the same period in 2024, driven by 31% year-over-year VOXZOGO revenue growth from new patients initiating therapy across all regions and the timing of large government orders, primarily in Latin America. In the quarter, revenues from BioMarin's Enzyme Therapies (ALDURAZYME®, BRINEURA®, NAGLAZYME®, PALYNZIQ and VIMIZIM®) also increased by 13% compared to the fourth quarter of 2024, driven by a combination of increased patient demand in all regions and the timing of large government orders.GAAP Net Loss was $47 million for the fourth quarter of 2025 compared to GAAP Net Income of $125 million for the same period in 2024. The increase in GAAP Net Loss was primarily due to the company's strategic decision to voluntarily withdraw ROCTAVIAN from the market resulting in charges of approximately $240 million during the quarter. These charges were mainly comprised of $119 million of an inventory write-off that was included in Cost of Sales and $118 million of long-lived asset impairments included in Selling, General and Administrative expense. The increase in GAAP Net Loss was partially offset by improved revenue growth as mentioned above and lower provision for income taxes.Non-GAAP Income for the fourth quarter of 2025 decreased to $89 million compared to $180 million for the same period in 2024. The decrease in Non-GAAP Income was primarily due to the ROCTAVIAN inventory write- off included in Cost of Sales. The decrease in Non-GAAP Income was partially offset by improved revenue growth as mentioned above. Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

Three Months EndedDecember 31,
Twelve Months EndedDecember 31,
2025
2024
% Change
2025
2024
% Change











Total Revenues$875
$747
17 %
$3,221
$2,854
13 %











Net Product Revenues by Product:










VOXZOGO$273
$208
31 %
$927
$735
26 %











Enzyme Therapies:










VIMIZIM$206
$191
8 %
$792
$740
7 %NAGLAZYME120
110
9 %
485
480
1 %PALYNZIQ125
100
25 %
433
355
22 %ALDURAZYME49
39
26 %
209
184
14 %BRINEURA49
48
2 %
186
169
10 %Total Enzyme Therapies Revenue$549
$488
13 %
$2,105
$1,928
9 %











KUVAN®$23
$28
(18) %
$100
$121
(17) %ROCTAVIAN$13
$11
18 %
$36
$26
38 %











GAAP Net Income (Loss) (1)$(47)
$125
(138) %
$349
$427
(18) %Non-GAAP Income (1)(2)$89
$180
(51) %
$614
$686
(10) %GAAP Operating Margin % (1)(3)(5.1) %
21.6 %


12.7 %
17.0 %

Non-GAAP Operating Margin % (1)(2)(5)15.1 %
31.1 %


23.3 %
28.6 %

GAAP Diluted Earnings (Loss) per Share (EPS)(1)(4)$(0.24)
$0.64
(138) %
$1.80
$2.21
(19) %Non-GAAP Diluted EPS (1)(2)(5)$0.46
$0.92
(50) %
$3.15
$3.52
(11) %

(1)Includes acquired IPR&D charges of $221 million (or approximately $1.10 on a per share basis) related to acquisition of Inozyme for the twelve months ended December 31, 2025.(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 (Loss) from Operations divided by Total Revenues.(4)Includes approximately $240 million of restructuring charges (or approximately $0.94 after tax on a per share basis) related to the company's strategic decision to voluntarily withdraw ROCTAVIAN from the market for the three and twelve months ended December 31, 2025.(5)Includes $119 million inventory write-off (or approximately $0.46 after tax on a per share basis) related to the company's strategic decision to voluntarily withdraw ROCTAVIAN from the market for the three and twelve months ended December 31, 2025.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.2026 Full-Year Financial Guidance (in millions, except EPS amounts)2026 guidance excludes any post-close contribution from the announced acquisition of Amicus Therapeutics, anticipated to close in Q2'26Total Revenues guidance reflects expectation of continued strong patient demand across Enzyme Therapies and VOXZOGO in 2026Other Revenue guidance reflects KUVAN, royalty revenue (including conclusion of U.S. Firdapse royalty term in January 2026), and the company's strategic decision to voluntarily withdraw ROCTAVIAN from the marketNon-GAAP Diluted EPS guidance includes approximately $0.25 of pre-close operating and interest expenses associated with the Amicus transactionIn 2026, excluding the impact of the Amicus transaction, Non-GAAP Operating Margin is expected to be approximately 40% for the full yearItem2025 Actuals
2026 GuidanceTotal Revenues$3,221
$3,325
to
$3,425Enzyme Therapies$2,105
$2,225
to
$2,275VOXZOGO$927
$975
to
$1,025Other Revenues(1)$189
$100
to
$125Non-GAAP Diluted EPS (2)(3)$3.15
$4.95
to
$5.15

(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.BioMarin will host a conference call and webcast to discuss fourth quarter 2025 financial results today, Monday, February 23, 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: 800-715-9871Replay Dial-in Number: 800-770-2030International Dial-in Number:  646-307-1963Replay International Dial-in Number: 609-800-9909Conference ID:  4503000 Conference ID: 4503000 About BioMarinBioMarin 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 multiple 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 StatementsThis 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 Operating Margin percentage, and Non-GAAP Diluted EPS for the full-year 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, the expected growth from VOXZOGO, and the expected impact of Other Revenues; the anticipated closing and benefits of BioMarin's proposed acquisition of Amicus Therapeutics, Inc.; 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 consummate and realize the anticipated benefits of any acquisitions; 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; 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 September 30, 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® and ROCTAVIAN® 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 INCOMEThree and Twelve Months Ended December 31, 2025 and 2024(In thousands of U.S. dollars, except per share amounts)(Unaudited)

Three Months EndedDecember 31,
Twelve Months EndedDecember 31,
2025
2024
2025
2024







REVENUES:






Net product revenues$              859,321
$              735,634
$           3,167,759
$           2,809,445Royalty and other revenues15,244
11,679
53,494
44,470Total revenues874,565
747,313
3,221,253
2,853,915OPERATING EXPENSES:






Cost of sales 275,709
136,139
717,442
580,235Research and development192,413
173,509
921,930
747,184Selling, general and administrative446,207
266,607
1,153,017
1,009,025Intangible asset amortization4,846
9,651
19,386
43,257Gain on sale of nonfinancial assets—
—
—
(10,000)Total operating expenses919,175
585,906
2,811,775
2,369,701INCOME (LOSS) FROM OPERATIONS(44,610)
161,407
409,478
484,214







Interest income19,210
17,680
74,904
74,883Interest expense(2,778)
(2,577)
(10,899)
(12,666)Other income (expense), net1,025
(6,871)
8,997
(4,668)INCOME (LOSS) BEFORE INCOME TAXES(27,153)
169,639
482,480
541,763Provision for income taxes19,420
44,696
133,579
114,904NET INCOME (LOSS)$              (46,573)
$              124,943
$              348,901
$              426,859EARNINGS (LOSS) PER SHARE, BASIC$                  (0.24)
$                    0.66
$                    1.82
$                    2.25EARNINGS (LOSS) PER SHARE, DILUTED$                  (0.24)
$                    0.64
$                    1.80
$                    2.21Weighted average common shares outstanding, basic192,225
190,688
191,787
190,027Weighted average common shares outstanding, diluted192,225
196,581
197,394
196,708 BIOMARIN PHARMACEUTICAL INC.CONDENSED CONSOLIDATED BALANCE SHEETSDecember 31, 2025 and 2024 (In thousands of U.S. dollars, except per share amounts)(Unaudited)

December 31, 2025
December 31, 2024ASSETS


Current assets:


Cash and cash equivalents$                    1,311,679
$                       942,842Short-term investments248,930
194,864Accounts receivable, net908,214
660,535Inventory1,298,883
1,232,653Other current assets185,784
201,533Total current assets3,953,490
3,232,427Noncurrent assets:


Long-term investments492,242
521,238Property, plant and equipment, net952,508
1,043,041Intangible assets, net213,837
255,278Goodwill196,199
196,199Deferred tax assets1,508,697
1,489,366Other assets277,049
251,391Total assets$                    7,594,022
$                    6,988,940LIABILITIES AND STOCKHOLDERS' EQUITY


Current liabilities:


Accounts payable and accrued liabilities$                       759,031
$                       606,988Total current liabilities759,031
606,988Noncurrent liabilities:


Long-term convertible debt, net597,176
595,138Other long-term liabilities150,816
128,824Total liabilities1,507,023
1,330,950Stockholders' equity:


Common stock, $0.001 par value: 500,000,000 shares authorized; 192,300,091 and
190,761,349 shares issued and outstanding, respectively192
191Additional paid-in capital5,956,582
5,802,068Company common stock held by the Nonqualified Deferred Compensation Plan(10,508)
(11,227)Accumulated other comprehensive income (loss)(13,473)
61,653Retained earnings (accumulated deficit)154,206
(194,695)Total stockholders' equity6,086,999
5,657,990Total liabilities and stockholders' equity$                    7,594,022
$                    6,988,940



 BIOMARIN PHARMACEUTICAL INC.CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWSTwelve Months Ended December 31, 2025 and 2024 (In thousands of U.S. dollars)(Unaudited)

Twelve Months Ended December 31,
2025
2024CASH FLOWS FROM OPERATING ACTIVITIES:


Net income$                348,901
$                426,859Adjustments to reconcile net income to net cash provided by operating activities:


Depreciation and amortization79,557
96,426Non-cash interest expense2,622
3,359Accretion of discount on investments(4,801)
(8,345)Stock-based compensation181,409
201,571Gain on sale of nonfinancial assets—
(10,000)Impairment of assets125,012
19,889ROCTAVIAN inventory write-off119,208
—Deferred income taxes48,738
56,096Unrealized foreign exchange gain4,459
(16,753)Acquired in-process research & development expense220,963
—Other(4,414)
20,135Changes in operating assets and liabilities:


Accounts receivable, net(228,054)
(57,909)Inventory(116,929)
(63,530)Other current assets8,891
(3,778)Other assets(38,573)
(73,700)Accounts payable and accrued liabilities66,136
(32,240)Other long-term liabilities14,869
14,761Net cash provided by operating activities827,994
572,841CASH FLOWS FROM INVESTING ACTIVITIES:


Purchases of property, plant and equipment(103,038)
(85,424)Maturities and sales of investments337,801
633,018Purchases of investments(355,875)
(410,250)Proceeds from sale of nonfinancial assets—
10,000Purchase of intangible assets(7,937)
(11,994)Acquisition, net of cash acquired(285,193)
—Other—
1,141Net cash provided by (used in) investing activities(414,242)
136,491CASH FLOWS FROM FINANCING ACTIVITIES:


Proceeds from exercises of awards under equity incentive plans14,460
49,277Taxes paid related to net share settlement of equity awards(55,965)
(77,560)Repayments of convertible debt—
(494,987)Other(889)
(3,177)Net cash used in financing activities(42,394)
(526,447)Effect of exchange rate changes on cash(2,521)
4,830NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS368,837
187,715Cash and cash equivalents:


Beginning of period$                942,842
$                755,127End of period$             1,311,679
$                942,842 Non-GAAP InformationThe 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 EndedDecember 31,
Twelve Months EndedDecember 31,
2025
2024
2025
2024







GAAP Reported Net Income (Loss)$             (47)
$             125
$             349
$             427Adjustments






Stock-based compensation expense - COS4
3
14
15Stock-based compensation expense - R&D14
14
55
60Stock-based compensation expense - SG&A29
34
113
127Amortization of intangible assets5
10
19
43Acquisition-related costs (2)—
—
15
—Gain on sale of nonfinancial assets (3)—
—
—
(10)Severance and restructuring costs (4)124
10
124
96Loss on investments (5)—
—
3
5Income tax effect of adjustments(40)
(16)
(78)
(76)Non-GAAP Income$               89
$             180
$             614
$             686 
Three Months EndedDecember 31,
Twelve Months EndedDecember 31,
2025
2024
2025
2024
R&D
SG&A
R&D
SG&A
R&D
SG&A
R&D
SG&A















GAAP expenses$          192
$          446
$         174
$          267
$          922
$      1,153
$          747
$      1,009Adjustments














Stock-based
compensation
expense(14)
(29)
(14)
(34)
(55)
(113)
(60)
(127)Acquisition-related
costs (2)—
—
—
—
—
(15)
—
—Severance and
restructuring costs (4)—
(124)
—
(10)
—
(124)
—
(96)Non-GAAP expenses$          178
$          292
$         159
$          222
$          867
$          901
$          688
$          786 
Three Months EndedDecember 31,
Twelve Months EndedDecember 31,
2025Percent
of GAAP
Total
Revenue
2024Percent
of GAAP
Total
Revenue
2025Percent
of GAAP
Total
Revenue
2024Percent
of GAAP
Total
Revenue











GAAP Income (Loss) from Operations$        (45)(5.1) %
$        16121.6 %
$        40912.7 %
$        48417.0 %Adjustments










Stock-based compensation expense475.4
516.8
1825.7
2027.1Amortization of intangible assets50.6
101.3
190.6
431.5Acquisition-related costs (2)——
——
150.5
——Gain on sale of nonfinancial assets (3)——
——
——
(10)(0.4)Severance and restructuring costs (4)12414.2
101.3
1243.8
963.4Non-GAAP Income from Operations$        13215.1 %
$        23231.1 %
$        75023.3 %
$        81528.6 % 
Three Months EndedDecember 31,


Twelve Months EndedDecember 31,
2025

2024


2025
2024










GAAP Diluted EPS$          (0.24)

$           0.64


$           1.80
$           2.21Adjustments









Stock-based compensation expense$           0.24

$           0.26


$           0.92
$           1.03Amortization of intangible assets$           0.03

$           0.05


$           0.10
$           0.22Acquisition-related costs (2)$               —

$               —


$           0.08
$               —Gain on sale of nonfinancial assets (3)$               —

$               —


$               —
$          (0.05)Severance and restructuring costs (4)$           0.63

$           0.05


$           0.63
$           0.49Loss on investments (5)$               —

$               —


$           0.02
$           0.03Income tax effect of adjustments$          (0.20)

$          (0.08)


$          (0.40)
$          (0.39)Non-GAAP Diluted EPS$           0.46

$           0.92

$           3.15
$           3.52

(1)Certain amounts may not sum or recalculate due to rounding.(2)These amounts were included in SG&A and represent severance costs incurred in the acquisition of Inozyme in July 2025.(3)Represents a payment triggered by a third party's attainment of a regulatory approval milestone related to previously sold intangible assets.(4)These amounts were included in SG&A and represent impairment of long-lived assets, severance and other restructuring costs related to the company's 2025 strategic decision to voluntarily withdraw ROCTAVIAN from the market and 2024 corporate initiatives and the associated organizational redesign efforts.(5)Represents impairment loss on non-marketable equity securities recorded in Other income (expense), net. 
Three Months EndedDecember 31,
Twelve Months EndedDecember 31,
2025
2024
2025
2024







GAAP Weighted-Average Diluted Shares Outstanding192.2
196.6
197.4
196.7Adjustments






Common stock issuable under the company's equity plans (1)0.8
—
—
—Common stock issuable under the Company's convertible debt(1)4.4
—
—
—Non-GAAP Weighted-Average Diluted Shares Outstanding197.4
196.6
197.4
196.7

(1)Common stock issuable under the company's equity plans and convertible debt were excluded from the computation of GAAP Weighted-Average Diluted Shares Outstanding for the three months ended December 31, 2025, as they were anti-dilutive. Contact:

Investors:
Media:Traci McCarty
Marni KottleBioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc.(415) 455-7558
(650) 374-2803 



View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-reports-fourth-quarter-and-full-year-2025-financial-and-operating-results-302694181.htmlSOURCE BioMarin Pharmaceutical Inc.

Original: BioMarin Reports Fourth Quarter and Full-year 2025 Financial and Operating Results

BioMarin, Ascendis slide as BridgeBio shakes up achondroplasia landscapeFebruary 12, 2026 10:25 AM
IH Market News
Shares of BioMarin Pharmaceutical (NASDAQ:BMRN) and Ascendis Pharma (NASDAQ:ASND) fell roughly 6% after rival BridgeBio Pharma (NASDAQ:BBIO) reported encouraging Phase 3 results for its experimental achondroplasia therapy.BridgeBio said its oral drug infigratinib achieved the primary endpoint in the late-stage PROPEL 3 trial, delivering superior annualized height velocity compared with placebo. The company cited a mean treatment difference of +2.10 cm per year, and noted the therapy also produced the first statistically significant improvement in body proportionality versus placebo in a randomized achondroplasia study.The update is viewed as a potential competitive challenge to BioMarin’s Voxzogo, currently the only treatment for achondroplasia approved by the U.S. Food and Drug Administration.Ascendis, meanwhile, is awaiting a regulatory decision on its once-weekly injectable therapy for achondroplasia, with potential FDA approval expected by February 28. Its shares also retreated following the BridgeBio announcement.Investors appear to be weighing the potential advantages of BridgeBio’s oral formulation, which showed strong efficacy and no serious adverse events linked to the study drug. The company plans to file regulatory submissions in the second half of 2026.Achondroplasia is a rare genetic disorder that affects bone growth, occurring in roughly one in 15,000 to 40,000 births. An estimated 55,000 people across the U.S. and European Union are living with the condition.BioMarin Pharmaceutical stock priceAscendis Pharma stock priceBridgeBio Pharma stock price

Original: BioMarin, Ascendis slide as BridgeBio shakes up achondroplasia landscape

BioMarin Announces Pricing of Private Offering of Senior Notes and Completion of Syndication of New Senior Secured Term Loan FacilityJanuary 29, 2026 4:05 PM
PR Newswire (US)

SAN RAFAEL, Calif., Jan. 29, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) ("BioMarin") announced today that it priced its previously announced offering of $850 million of 5.500% senior unsecured notes due 2034 (the "Notes"). The issue price of the Notes is 100.000%. The offering is expected to close on February 12, 2026, subject to the satisfaction of customary closing conditions.







BioMarin also announced that, in connection with the pending acquisition (the "Acquisition") of Amicus Therapeutics, Inc. ("Amicus"), it completed the syndication of a new $2 billion senior secured term loan "B" facility (the "Term Loan B Facility"), which Term Loan B Facility is in addition to a $800 million senior secured term loan "A" facility (the "Term Loan A Facility" and, together with the Term Loan B Facility, the "Term Facilities"), and a $600 million senior secured revolving credit facility into which BioMarin expects to enter in connection with the Acquisition (the "New Revolving Facility" and, together with the Term Facilities, the "New Senior Secured Credit Facilities").BioMarin intends to use the net proceeds from the offering of the Notes, together with borrowings under the Term Facilities and cash on hand, to fund the consideration payable in connection with the Acquisition and related fees and expenses in connection with the Acquisition, the borrowings under the New Senior Secured Credit Facilities, and the issuance of the Notes. The company may also borrow up to $150 million under the New Revolving Facility to pay such fees and expenses.Gross proceeds from the issuance of the Notes will be deposited into an escrow account at the closing of the Offering, pending consummation of the Acquisition. In the event that the Acquisition is not completed on or prior to December 19, 2026, or upon the occurrence of certain other events, BioMarin will be required to redeem all of the Notes at a redemption price equal to 100% of the initial issue price of the Notes plus accrued and unpaid interest from the date of issuance, or the most recent date to which interest has been paid or provided for, to but excluding the special mandatory redemption date.The Notes will be jointly and severally guaranteed by certain of BioMarin's subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities, including, after the closing of the Acquisition, Amicus and certain of its subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities.The indenture governing the Notes is expected to contain customary covenants that, among other things, restrict, with certain exceptions, the ability of each of BioMarin and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets.The Notes have not been, and will not be, registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other securities laws and may not be offered or sold in the United States absent an effective registration statement or an applicable exemption from the registration requirements of or in a transaction not subject to the Securities Act and any state or other applicable securities laws. Accordingly, the offering of the Notes is available only to a limited number of persons who are either (1) reasonably believed to be "qualified institutional buyers" as defined in Rule 144A under the Securities Act or (2) non-U.S. persons outside the United States pursuant to Regulation S under the Securities Act. The Notes will be subject to restrictions on transferability and resale and may not be transferred or resold except in compliance with the registration requirements of the Securities Act or pursuant to an exemption therefrom and in compliance with any state or other applicable securities laws.This press release is for information purposes only and is not intended to and does not constitute, or form part of, an offer, invitation or the solicitation of an offer or invitation to purchase, otherwise acquire, subscribe for, sell or otherwise dispose of any securities, or the solicitation of any vote or approval in any jurisdiction, pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. This press release contains information about the pending offering of the Notes, and there can be no assurance that the offering will be completed. The offering of the Notes may be made only by means of an offering memorandum.About BioMarinBioMarin 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 eight 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.Forward-Looking StatementsThis press release contains forward-looking statements about the proposed offering of the Notes, the entry into the New Senior Secured Credit Facilities, BioMarin's intention to issue the Notes, and the expected use of proceeds. 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 timing to consummate the proposed Notes offering, New Senior Secured Credit Facilities and Acquisition, and the risk that such transactions may not close, as well as 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 September 30, 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, except as required by law.BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.Contacts:
InvestorsMediaTraci McCarty  Erin RauBioMarin Pharmaceutical Inc.  BioMarin Pharmaceutical Inc.(415) 455-7558  (925) 683-9622 



View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-pricing-of-private-offering-of-senior-notes-and-completion-of-syndication-of-new-senior-secured-term-loan-facility-302674465.htmlSOURCE BioMarin Pharmaceutical Inc.

Original: BioMarin Announces Pricing of Private Offering of Senior Notes and Completion of Syndication of New Senior Secured Term Loan Facility

BioMarin Announces Proposed Private Offering of Senior Notes and Syndication of New Senior Secured Term Loan FacilityJanuary 26, 2026 12:45 PM
PR Newswire (US)

SAN RAFAEL, Calif., Jan. 26, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) ("BioMarin") announced today that it intends to offer, subject to market and other conditions, $850 million of senior unsecured notes due 2034 (the "Notes").







BioMarin also announced that, in connection with the pending acquisition (the "Acquisition") of Amicus Therapeutics, Inc. ("Amicus"), it launched the syndication of a new $2 billion senior secured term loan "B" facility (the "Term Loan B Facility"), which Term Loan B Facility is in addition to a $800 million senior secured term loan "A" facility (the "Term Loan A Facility" and, together with the Term Loan B Facility, the "Term Facilities"), and a $600 million senior secured revolving credit facility into which BioMarin expects to enter in connection with the Acquisition (the "New Revolving Facility" and, together with the Term Facilities, the "New Senior Secured Credit Facilities").BioMarin intends to use the net proceeds from the offering of the Notes, together with borrowings under the Term Facilities and cash on hand, to fund the consideration payable in connection with the Acquisition and related fees and expenses in connection with the Acquisition, the borrowings under the New Senior Secured Credit Facilities, and the issuance of the Notes. The company may also borrow up to $150 million under the New Revolving Facility to pay such fees and expenses.Gross proceeds from the issuance of the Notes will be deposited into an escrow account at the closing of the Offering, pending consummation of the Acquisition. In the event that the Acquisition is not completed on or prior to December 19, 2026, or upon the occurrence of certain other events, BioMarin will be required to redeem all of the Notes at a redemption price equal to 100% of the initial issue price of the Notes plus accrued and unpaid interest from the date of issuance, or the most recent date to which interest has been paid or provided for, to but excluding the special mandatory redemption date.The Notes will be jointly and severally guaranteed by certain of BioMarin's subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities, including, after the closing of the Acquisition, Amicus and certain of its subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities.The indenture governing the Notes is expected to contain customary covenants that, among other things, restrict, with certain exceptions, the ability of each of BioMarin and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets.The Notes will not be registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other securities laws and may not be offered or sold in the United States absent an effective registration statement or an applicable exemption from the registration requirements of or in a transaction not subject to the Securities Act and any state or other applicable securities laws. Accordingly, the offering of the Notes is available only to a limited number of persons who are either (1) reasonably believed to be "qualified institutional buyers" as defined in Rule 144A under the Securities Act or (2) non-U.S. persons outside the United States pursuant to Regulation S under the Securities Act. The Notes will be subject to restrictions on transferability and resale and may not be transferred or resold except in compliance with the registration requirements of the Securities Act or pursuant to an exemption therefrom and in compliance with any state or other applicable securities laws.This press release is for information purposes only and is not intended to and does not constitute, or form part of, an offer, invitation or the solicitation of an offer or invitation to purchase, otherwise acquire, subscribe for, sell or otherwise dispose of any securities, or the solicitation of any vote or approval in any jurisdiction, pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. This press release contains information about the pending offering of the Notes, and there can be no assurance that the offering will be completed. The offering of the Notes may be made only by means of an offering memorandum.About BioMarinBioMarin 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 eight 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.Forward-Looking StatementsThis press release contains forward-looking statements about the proposed offering of the Notes, the entry into the New Senior Secured Credit Facilities, BioMarin's intention to issue the Notes and the expected use of proceeds. 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 timing to consummate the proposed Notes offering, New Senior Secured Credit Facilities and Acquisition, and the risk that such transactions may not close, as well as 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 September 30, 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, except as required by law.BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.Contacts:
Investors                                                                                    MediaTraci McCarty                                                                             Erin RauBioMarin Pharmaceutical Inc.                                                  BioMarin Pharmaceutical Inc.(415) 455-7558                                                                 (925) 683-9622



View original content to download multimedia:https://www.prnewswire.com/news-releases/biomarin-announces-proposed-private-offering-of-senior-notes-and-syndication-of-new-senior-secured-term-loan-facility-302670007.htmlSOURCE BioMarin Pharmaceutical Inc.

Original: BioMarin Announces Proposed Private Offering of Senior Notes and Syndication of New Senior Secured Term Loan Facility

BMRN 10 Q due 2/19

BMRN volatile stock

GRABAR LAW OFFICE INVESTIGATES POTENTIAL DERIVATIVE ACTION ON BEHALF OF CURRENT SHAREHOLDERS OF BIOMARIN PHARMACEUTICAL INC. (NASDAQ: BMRN)
A class action alleging BioMarin misled investors survived a motion to dismiss. For more information see https://grabarlaw.com/the-latest/biomarin-pharma-investigation/

and the CEO gets on a earnings call and lowers guidance for Q4

and that is that

crushed the expectations on EPS

options trading on BMRN is insane

BMRN shorts will be “suprised and burned” by CHMP - will be SO NICE.

Hey Shorts-why didn’t the FDA inspect BMRN Valrox factory in Nevada, while the EMA inspected the BMRN Valrox factory in Ireland 3 months ago? Actions speak louder than words - FDA had no intention of Approving without more data and EMA does - Ask BA CSI Wedbush and Cantor.

BMRN
Cantor Fitzgerald Note on BMRN 8/19- Price Target $125 from $129 - Overweight maintained:

“We think the base business alone (NPV, removing spend for pipeline) is worth $90/sh vs. shares at $76/sh currently. While CRL is a setback, we still see significant growth ahead for BMRN. We see potential for upside from execution across EU Roctavian review, vosoritide (achondroplasia, regulatory submissions accepted), and emerging gene therapy portfolio, coupled with manufacturing expertise. We note that the PKU gene therapy program is expected to dose its first patient this quarter and we think shares reflect limited credit to that program.”

BMRN
Wedbush analyst Liana Moussatos reiterated a Buy rating on BioMarin Pharmaceuticals-
– Research Report) today and set a price target of $136.00. The company’s shares closed last Wednesday at $75.04.

According to tipranks.com, Moussatos is a 5-star analyst with an average return of 12.2% and a 53.4% success rate.

CMA for BMRN - BMRN will receive a “Conditional marketing authorisation“ -for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A-
“The approval of a medicine that address unmet medical needs of patients on the basis of less comprehensive data than normally required. The available data must indicate that the medicine’s benefits outweigh its risks and the applicant should be in a position to provide the comprehensive clinical data in the future.”

https://www.ema.europa.eu/en/glossary/conditional-marketing-authorisation

EMA will ignore Trumps FDA -greater Hemophilia need :
BA Analyst Meachum says we can still see EU
$BMRN
approval in 2020- EMA says 210 days til word on
$BMRN
Hemophilia approval - wow - even with clock stops - any day now : SAN RAFAEL, Calif., Dec. 23, 2019 /PRNewswire/ -- “BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January 2020 under accelerated assessment.

EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 and recently granted BioMarin's request for accelerated assessment of this MAA-This submission marks the first marketing application submission in Europe for a gene therapy product for any type of hemophilia.
Evaluating a MAA under the EMA centralized procedure can take up to 210 days.”

BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia

Published: Aug 20, 2020

= “near term growth “- as is pending Hemophilia approval in EU - a larger market for Hemophiia than is the US

https://www.biospace.com/article/releases/biomarin-submits-new-drug-application-to-u-s-food-and-drug-administration-for-vosoritide-to-treat-children-with-achondroplasia/

BMRN
Sorry CitI - BA analyst Geoff Meachum (maintains Buy with $105 price target) and Canaccord Genuity analyst Michelle Gilson say you’re wrong-Gilson maintains a Buy rating on BioMarin Pharmaceutical yesterday and set a price target of $95.00.

According to tipranks.com/, Gilson is a 5-star analyst with an average return of 35.1% and a 63.8% success rate.

Bank America note on BMRN

”Approval still in the cards...just delayed
-perhaps 2 years“
Reiterate Rating: BUY | PO: 105.00 USD

No accelerated approval for $SGMO $PFE on 4/6 WEEK followup - $BMRN has triggered 2 year followup for all- Lead maintained.
https://endpts.com/sangamo-pfizer-are-catching-up-to-biomarin-and-spark-on-a-gene-therapy-for-hemophilia-a-with-a-long-way-to-go/

$BAC On $BMRN

“FDA has requested BioMarin provide 2-year annualized bleed rate (ABR) data for Roctavian to complete the filing package (which BioMarin expects to have in November 2021). 6-month phase 3 data showed 85%/100% reduction in mean/median ABR and 4- year phase 1/2 data showed a 95%/100% reduction, which gives us comfort in the reproducibility in 2-year phase 3 data. Notably, in the phase 1/2 dataset, the 2-year mean bleed rate improvement was better than in year 1 (99% vs. 92%) so we could see the 85% reduction potentially improve. If Roctavian can show anything near this in November 2021, we think BioMarin should have no problem getting approval.”

Ttttttiiiiiimmmmmmmbbbbbeeeerrrrr!!!!!!!

$131.19 and still climbing

93.22

talk about missing the boat, I had an alert go off a couple of weeks ago on a price dip below 69.00

I hadn't been keeping up with BMRN lately so I blew it off

now trading at 84.50

I guess I put that alert on this puppy for a reason

Experiments in juveniles and infants can garner public praise, lower regulatory hurdles, and mask overspending. It creates a unique and problematic bias. In the US, mores are well established in this regard. One cannot question the utility of such treatments without being vilified.

Two catalysts this month for BMRN.
1. PDUFA
2. Hem A data

Here is the info link for upcoming biotech data insert-text-here

Thanks

Out @ $88.50 for a small profit after buying down from a too high entry. Probably a multi-day runner, but changing brokers (leaving Ally Invest which has been terrible) and had to sell.

Could be back in tomorrow if transfer proceeds well.

BMRN

very nice move off of the bottom here. I was holding out to start taking a starter until 80.00... good to hold discipline... but I missed a nice move

According to WhaleWisdom, BMRN is Baker Bro 6th largest holding with 22% increase in shares as of the last filing:

BMRN HEALTH CARE 5,905,673 $ 536,353,000 2.59% 3.45% 6 1,099,190 22% 3.4163% Q2 2012 13F 2017-06-30 2017-08-14

https://whalewisdom.com/filer/baker-bros-advisors-llc#/tabholdings_tab_link

BMRN

Sarepta Therapeutics and BioMarin Pharmaceutical Inc. Announce Execution of a Global Settlement and a License Agreement Resolving Exon Skipping Patent Litigation

Agreement terms resolve global patent proceedings regarding Sarepta’s sale of EXONDYS 51® (eteplirsen) and future Duchenne muscular dystrophy (DMD) exon-skipping products

July 18, 2017 08:30 ET | Source: Sarepta Therapeutics
CAMBRIDGE, Mass. and SAN RAFAEL, Calif., July 18, 2017 (GLOBE NEWSWIRE) --

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a leading biotechnology company in therapies for rare genetic diseases, announced today that Sarepta and BioMarin executed a license agreement that provides Sarepta Therapeutics with global exclusive rights to BioMarin’s DMD patent estate for EXONDYS 51 and all future exon-skipping products. BioMarin retains the right to convert the license to a co-exclusive right in the event it decides to proceed with an exon-skipping therapy for DMD. In addition, Sarepta and BioMarin executed a settlement agreement, resolving the ongoing worldwide patent proceedings related to the use of EXONDYS 51 and all future exon-skipping products for the treatment of DMD. The effectiveness of the agreements is subject to closing conditions including execution of necessary approvals by Academisch Ziekenhuis Leiden (AZL) by July 24, 2017.

Under the terms of the license and settlement agreements, Sarepta will make a one-time payment of $35 million to BioMarin and certain additional regulatory and commercial milestone payments for exons 51, 45, 53 and possibly on future exon-skipping products.

In addition, Sarepta will pay royalties to BioMarin as follows:

Exon-skipping compounds 51, 45, and 53 and possibly on future exon-skipping products: Sarepta will pay BioMarin 5 percent of net sales through the end of 2023 in the United States; and
Exon-skipping compounds 51, 45, and 53 and possibly on future exon-skipping products: Sarepta will pay BioMarin 8 percent of net sales through September 30, 2024 in the European Union and in other countries where certain BioMarin / AZL patents exist.
“Upon their effectiveness, these global license and settlement agreements provide Sarepta worldwide freedom to operate for EXONDYS 51 and our future exon-skipping products,” said Douglas Ingram, Sarepta’s President and Chief Executive Officer. “The resolution of these legal matters provides us with more certainty to fully focus our resources and energy on our crucial mission of developing innovative medicines to improve the lives of those impacted by DMD around the world."

“We are pleased to reach a global settlement and license agreement with Sarepta that fairly recognizes the important innovation by the Leiden University Medical Center and allows patients certainty that this issue will not create a barrier to access,” said G. Eric Davis, BioMarin’s Executive Vice President and General Counsel.

About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of EXONDYS 51 in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51 has not been established.

Important Safety Information
Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51 infusion.

About Sarepta Therapeutics
Sarepta Therapeutics is a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases. The company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.

About BioMarin Pharmaceutical Inc.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statements
This press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements about the license agreement providing Sarepta with global exclusive rights to BioMarin’s DMD patent estate for EXONDYS 51 and all future exon-skipping products; the settlement agreement resolving the ongoing worldwide patent proceedings related to the use of EXONDYS 51 and all future exon-skipping products for the treatment of DMD; the payments and royalties that Sarepta will be making as part of the settlement and license agreements; the settlement and license agreements providing for Sarepta's worldwide freedom to operate for EXONDYS 51 and Sarepta’s future exon-skipping products; the settlement providing Sarepta with the certainty to fully focus its resources and energy on its crucial mission of developing innovative medicines to improve the lives of those impacted by DMD around the world; and the statement that the patent proceedings between the parties will not create for patients a barrier to access to the innovation by the Leiden University Medical Center.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: the settlement and license agreements may not become effective if their conditions to effectiveness are not met within the required deadline; the parties may not be able to fulfill their commitments and obligations under the settlement and license agreements; any future claims of infringement by other third parties; the expected benefits and opportunities related to the settlement and license agreements between the parties may not be realized or may take longer to realize than expected due to challenges and uncertainties regarding the sales of EXONDYS 51 and the research and development of future exon-skipping products; Sarepta may experience significant fluctuations in sales of EXONDYS 51 from period to period and, ultimately, Sarepta may never generate sufficient revenues from EXONDYS 51 to reach or maintain profitability or sustain its anticipated levels of operations; Sarepta may never receive regulatory approval to its future exon-skipping products due to a variety of reasons including that the results of additional research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates; and even if Sarepta obtains regulatory approvals, it may not achieve any significant revenues from the sale of such products; Sarepta may not have worldwide freedom to operate for EXONDYS 51 and Sarepta’s future exon-skipping products due to future proceedings brought by other parties.

Any of the foregoing risks could adversely affect Sarepta's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2016 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2017 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
or
Investors:
BioMarin Pharmaceutical Inc.
Traci McCarty, 415-455-7558
Media:
BioMarin Pharmaceutical Inc.
Debra Charlesworth, 415-455-7451
______________________________________________

BMRN

Wrong choice. Hugh volume today and now near the 3 month low on no news and biotech up marginally.

BMRN

In BMRN @ $88.80 today. Hadn't traded it for over 3 years.

A Baker Bro stock with limited up potential:

Consensus Ratings for BioMarin Pharmaceutical (NASDAQ:BMRN) (How are Consensus Ratings Calculated?)
Ratings Breakdown: 6 Hold Ratings, 14 Buy Ratings
Consensus Rating: Buy (Score: 2.70)
Consensus Price Target: $113.61 (24.85% upside)
_______________________________________

https://www.marketbeat.com/stocks/NASDAQ/BMRN/

https://seekingalpha.com/article/4087040-biomarin-hemophila-candidates-looks-impress-community

Always good to see the bulls.

It's a ways off, but Jan 27 will be a good day if/when new drug gets approved. I see that sometimes FDA approves on a date prior to the scheduled date - does anyone know if that sort of news that a decision is going to be made early (ie if approval/CRL) is avail before it actually happens?

BMRN bullish 91.99

optionable

FDA denied BMRN. SRPT is leading now.

BioMarin, Sarepta’s Loss Could Be PTC Therapeutic’s Gain

http://marketexclusive.com/biomarin-sareptas-loss-could-be-ptcs-gain/1419/

Could the FDA Approve Both BioMarin and Sarepta’s DMD Candidates?

http://marketexclusive.com/could-the-fda-approve-both-biomarin-and-sareptas-dmd-candidates/1184/

Two Stocks to Watch This Month in Biotech - article discusses BioMarin http://marketexclusive.com/two-stocks/1128/

Surprised to see you here. Bio-Marin is heading back up FYI ..

Looks like we're all going to be on the sidelines today (BMRN also had positive news from the UK's Health Technology Assessment agency, NICE, today, (http://www.pharmatimes.com/Article/15-11-23/NICE_draft_yes_for_BioMarin_s_Vimizim.aspx) but that will likely be drowned out by the outcome of today's FDA review meeting).

"BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Announced Today the Stock is Halted

Date : 11/24/2015 @ 7:05AM
Source : GlobeNewswire Inc.
Stock : Biomarin Pharmaceutical Inc. (MM) (BMRN)

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) Announced Today the Stock is Halted


Today : Tuesday 24 November 2015


BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that NASDAQ has halted trading of the company’s stock. The Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) of the U.S. Food and Drug Administration (FDA) is meeting today to review BioMarin’s New Drug Application (NDA) for Kyndrisa, an investigational antisense oligonucleotide drug candidate for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.


The PCNS advisory meeting is scheduled for November 24 at 8:00 a.m. to 5:30 p.m. EST. The briefing materials and webcast information can be found on the FDA website at: www.fda.gov/AdvisoryCommittees/Calendar/ucm467180.htm

The Prescription Drug User Fee Act (PDUFA) action date for completion of FDA review of the Kyndrisa NDA is December 27, 2015.

About Duchenne Muscular Dystrophy

Changes in the dystrophin gene (mutations) that lead to the near absence of dystrophin protein result in the most severe form of dystrophin deficient muscular dystrophy, Duchenne muscular dystrophy, also known as just Duchenne. Boys living with Duchenne experience progressive muscle weakness, causing serious medical complications including serious heart or respiratory-related complications, resulting in death in early adulthood.

Primarily affecting boys, Duchenne affects approximately 1 in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood. There is currently no FDA approved therapy designed specifically to treat Duchenne.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of five commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.BMRN.com.
Investors
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558

Media
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451"