BioMarin Pharmaceutical Inc. is a biotechnology company that develops and commercializes therapies for life-threatening rare diseases and medical conditions. It operates in the United States, Europe, Latin America, the Middle East, the Asia Pacific, and other international markets. Its products include VIMIZIM, VOXZOGO, NAGLAZYME, and PALYNZIQ, and it is developing BRINEURA, ALDURAZYME, KUVAN, BMN 333, and BMN 351. The company serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers, and has license and collaboration agreements with Ares Trading S.A. BioMarin was incorporated in 1996 and is based in San Rafael, California.
BioMarin Targets $200 Million Amicus Synergies as VOXZOGO Nears $1 Billion
BioMarin Pharmaceutical said it is progressing with the integration of Amicus Therapeutics and expects $200 million in annual non-GAAP cost savings, with most synergies realized in 2027 and full realization in 2028. Speaking at Morgan Stanley's Global Healthcare Conference, President and CEO Alexander Hardy said Amicus had about 505 employees and BioMarin expects to retain approximately 192 over the long term, with about 70% of the savings coming from general and administrative expenses and the remainder from duplicated research and development functions. Chief Financial Officer Brian Mueller said the deal should be accretive in its first year, with substantial accretion beginning next year, and that leverage is now expected to fall below 2.5 times by the middle of 2027, nearly a year earlier than the initial target. Hardy said VOXZOGO delivered 20% quarterly patient growth despite U.S. competition and is expected to reach $1 billion in revenue this year, which would make it BioMarin's first blockbuster product, while a potential FDA approval in hypochondroplasia is not incorporated into its 2026 outlook. BioMarin projects peak sales of approximately $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI + OPFOLDA by the mid-2030s, and said PALYNZIQ grew 27% to $135 million while VIMIZIM has the potential to exceed $1 billion in revenue.
FDA Grants Priority Review to AstraZeneca's Efzimfotase Alfa Filing for Rare Bone Disease HPP
AstraZeneca said the FDA accepted its regulatory filing for the investigational enzyme replacement therapy efzimfotase alfa in patients aged two years and older with hypophosphatasia, or HPP, and granted the application priority review, shortening the review period by four months, with a final decision expected during the first half of 2027. The filing is supported by data from three phase III studies: MULBERRY met its primary endpoint in treatment-naive children aged two to less than 12 years old, showing a significant improvement in bone health, and CHESTNUT showed that patients switching from Strensiq could maintain the benefits of treatment, while HICKORY did not meet its primary endpoint in treatment-naive individuals aged 12 years and older, though AstraZeneca reported a numerical improvement, particularly in patients with pediatric-onset disease. The company said efzimfotase alfa was generally well-tolerated with an acceptable safety profile across the three studies. The commercial angle rests on Strensiq, the established HPP treatment AstraZeneca gained through its 2021 acquisition of Alexion, which was approved by the FDA in 2015 as the first bone-targeted enzyme replacement therapy for HPP and generated $1.05 billion in sales in the first half of 2026, up 41% year over year. Efzimfotase alfa is designed to require lower injection volumes and substantially less frequent dosing, once every two weeks, compared with Strensiq's three- or six-times-weekly regimen. Elsewhere in the space, BioMarin Pharmaceutical entered HPP through its acquisition of Alesta Therapeutics, completed earlier this month, paying $275 million upfront with up to $215 million in additional milestone payments for ALE1, an oral small-molecule therapy in a phase I/IIa study, while Recursion Pharmaceuticals is developing the oral ENPP1 inhibitor REC-102, formerly REV102, which remains in IND-enabling studies with a data-driven decision on a phase I study expected before the end of this year.
BioMarin Appoints Bristol Myers Squibb Research Chief Robert Plenge to Board
BioMarin Pharmaceutical Inc. has appointed Robert Plenge, M.D., Ph.D., to its Board of Directors, effective September 10, 2026. Dr. Plenge is Executive Vice President, Chief Research Officer and Head of Research at Bristol Myers Squibb, where he leads scientific work across nine research sites worldwide. He joined Bristol Myers Squibb in November 2019 through its acquisition of Celgene Corp., where he served as vice president of the immunology and inflammation portfolio in research and early development, and previously was vice president and head of translational medicine at Merck & Co. BioMarin President and Chief Executive Officer Alexander Hardy said Dr. Plenge will bring an important scientific perspective as the company develops its research capability and expands its pipeline in genetic conditions. Dr. Plenge also serves on the boards of Alltrna and the PhRMA Foundation, and received his M.D. and Ph.D. from Case Western Reserve University.
BioMarin and Ascendis Settle Global Patent Dispute Over Yuviwel
BioMarin Pharmaceutical and Ascendis Pharma have entered into binding agreements to resolve their global patent dispute over Ascendis's achondroplasia drug Yuviwel, sending BioMarin shares up 5% on August 31. Under the settlement, Ascendis will pay BioMarin a royalty equal to 20% of Yuviwel's net sales in the US, retroactive to the drug's first commercial sale, and 18% of net sales in the European Union, Brazil, and South Korea, with payments running through May 20, 2030. In exchange, BioMarin will grant Ascendis a non-exclusive, worldwide, royalty-bearing license for navepegritide, Yuviwel's active compound, allowing Ascendis to continue studying, developing, manufacturing, and commercializing the medication without restrictions. The deal resolves litigation pending in numerous jurisdictions, including a Section 337 investigation by the US International Trade Commission that BioMarin has agreed to drop. BioMarin CEO Alexander Hardy said the settlement encourages continued long-term investment in breakthrough treatments, while Ascendis CEO Jan Mikkelsen said the terms reflect Yuviwel's differentiated profile and the significant unmet medical need it addresses.
Achondroplasia Market to Reach $600M in 2025, Growing 11% CAGR to 2036
The achondroplasia market across the seven major markets—the United States, Germany, France, Italy, Spain, the United Kingdom, and Japan—was valued at approximately USD 600 million in 2025 and is expected to grow at a compound annual growth rate of approximately 11% from 2026 to 2036, according to a new report from ResearchAndMarkets.com. The diagnosed prevalent population in these markets was estimated at approximately 28,500 cases in 2025, with the United States representing the largest patient population and commercial market at approximately 14,500 cases. The treatment landscape is shifting toward targeted therapies, with approved drugs VOXZOGO (vosoritide) and YUVIWEL (navepegritide; TransCon CNP) leading the way, while emerging pipeline candidates include infigratinib (BBP-831/BGJ398) from QED Therapeutics, a BridgeBio company, in collaboration with Novartis and Kyowa Kirin, RBM-007 (umedaptanib pegol) from RIBOMIC, and BMN 333 from BioMarin Pharmaceutical. Recent developments include RIBOMIC's Phase II summary and Phase III application in Japan, BridgeBio's positive Phase III PROPEL 3 results for infigratinib, and BioMarin's Phase I data for BMN 333. The report highlights opportunities in earlier genetic diagnosis, targeted FGFR3 therapies, and long-acting CNP analogs, while noting unmet needs such as limited treatment options across age groups and high treatment costs.
BioMarin Secures 20% U.S. Royalties on Yuviwel in Patent Settlement
BioMarin Pharmaceutical has converted its global patent dispute with Ascendis Pharma into a royalty-bearing license for Yuviwel, a once-weekly achondroplasia treatment, resolving proceedings before the U.S. International Trade Commission and courts in several countries. Under the settlement, BioMarin will receive royalties equal to 20% of U.S. Yuviwel net sales, retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil, and South Korea through May 2030. The agreement eliminates litigation costs and uncertainty while giving BioMarin a share of Yuviwel's economics without funding its development or commercialization. However, the royalties may only partially offset cannibalization of BioMarin's Voxzogo franchise, which is expected to generate $1.0 billion to $1.05 billion in 2026 revenue. Ascendis reported €8 million in second-quarter 2026 Yuviwel revenue and over 220 unique U.S. patient enrollments through July 31, with more than 65% approved for reimbursement. The license also covers patents related to all current and potential Yuviwel indications, including hypochondroplasia and combination therapies, reducing BioMarin's future blocking leverage. The financial outcome depends on whether Yuviwel expands the treated population or primarily displaces Voxzogo sales.
BioMarin Completes Acquisition of Alesta Therapeutics
BioMarin Pharmaceutical Inc. announced today that it has completed its acquisition of Alesta Therapeutics, adding ALE1, a potential first oral therapy for hypophosphatasia, to its clinical pipeline. The deal, first announced on August 18, strengthens BioMarin's skeletal conditions portfolio with ALE1, an oral small molecule currently in a Phase 1/2a trial for HPP, a rare genetic bone disease. Prior to closing, Alesta spun off all non-ALE1 assets, and Alesta is now a wholly owned subsidiary of BioMarin. BioMarin, a San Rafael, California-based rare disease biotech with nine commercial therapies, expects the acquisition to bolster its pipeline of category-defining medicines.
BioMarin Pharmaceutical to Acquire Alesta Therapeutics for $275 Million
BioMarin Pharmaceutical agreed to acquire clinical-stage biotech Alesta Therapeutics for $275 million upfront, with up to $215 million in additional milestone payments. The deal gives BioMarin access to Alesta's lead asset ALE1, a potential oral therapy for the rare genetic bone disease hypophosphatasia. Barclays raised its price target on BioMarin to $111 from $105, and Piper Sandler lifted its target to $88 from $82, both maintaining Overweight ratings, while H.C. Wainwright lowered its target to $59 from $60 with a Neutral rating. BioMarin shares closed at $69.33, up 3.5%.
BioMarin Raises 2026 Revenue Outlook and Files ESOP Shelf
BioMarin Pharmaceutical raised its full-year 2026 revenue guidance to between US$3.88 billion and US$3.93 billion and filed a US$719.24 million common stock shelf registration for an ESOP-related offering. The company reported second-quarter 2026 revenue of US$989.71 million and provided more detailed third and fourth quarter expectations, with the higher outlook leaning on a stronger fourth quarter and Amicus contribution. The raised guidance modestly reinforces the near-term growth catalyst but does little to reduce the key risk around rising costs outpacing revenue and the potential need for further dilutive financing if performance weakens. Some of the lowest analysts were already cautious, assuming about US$4.5 billion revenue and US$752.9 million earnings by 2029, focusing heavily on pricing and reimbursement risk.
BioMarin Pharmaceutical Updates Guidance and Files Shelf Registration
BioMarin Pharmaceutical has updated its guidance and filed a shelf registration, alongside reporting second quarter 2026 results. The company's share price stands at US$67.36, with a 90-day return of 35.62% and a 1-year total shareholder return of 17.07%, though the 3-year total shareholder return is down 24.36%. Analysts expect earnings to reach $1.3 billion, or $6.72 per share, by about June 2029, up from $268.7 million today, with estimates ranging from $759.4 million to $2.1 billion. A discounted cash flow analysis using a 7.32% discount rate suggests a fair value of $87.85, implying the stock is 23.3% undervalued.
Nearly nine in ten EU4+UK hematologists now require longer-term efficacy data before referring patients with hemophilia A for gene therapy, according to new research from Spherix Global Insights. The 2026 Market Dynamix study of 160 hematologists across France, Germany, Italy, Spain, and the United Kingdom found that 89% of physicians need longer-term efficacy data, up 23 percentage points from 66% in 2025. Patient research shows 37% of 169 US patients with hemophilia A or B were considered good candidates for gene therapy but were not currently pursuing it, while 36% of hemophilia A patients and 24% of hemophilia B patients expressed strong interest. The findings come amid commercial setbacks including the discontinuation of BEQVEZ by Pfizer, withdrawal of ROCTAVIAN by BioMarin, and supply challenges for HEMGENIX by CSL Behring.
BioMarin Pharmaceutical's VOXZOGO FDA Filing Accepted, Decision by February 2027
The FDA has accepted BioMarin Pharmaceutical's supplemental New Drug Application for VOXZOGO, setting a target decision date of February 28, 2027. The stock has seen a 5.81% return over the past 30 days and a 6.72% return over 90 days, though the three-year total shareholder return remains down 31.69%. A widely followed narrative values the company at a fair value of $87.85 per share, implying a 33% upside from the last close of $59.20, driven by strong revenue growth from therapies like VOXZOGO and VIMIZIM, pipeline advances including late-stage programs for achondroplasia and ENPP1 deficiency, and expanding addressable markets through improved genetic diagnosis. However, the current price-to-earnings ratio of 42.6 times is above both the US biotech industry average of 17.4 times and a fair ratio of 27.5 times, indicating valuation risk if sentiment shifts.
FDA accepts BioMarin sNDA for full Voxzogo approval
The U.S. Food and Drug Administration has accepted BioMarin Pharmaceutical's supplemental New Drug Application seeking full approval of its achondroplasia treatment Voxzogo. The FDA set an action date of February 28, 2027. The application is supported by three long-term trials. Voxzogo was granted accelerated approval in 2021.
Therapeutics stocks beat Q1 revenue estimates by 14.5%
The 11 therapeutics stocks tracked by this publication reported a satisfactory first quarter, with aggregate revenues beating analysts' consensus estimates by 14.5%. Myriad Genetics posted revenues of $200.4 million, up 2.3% year on year but missing expectations by 1%, while Moderna delivered the fastest revenue growth among its peers with $389 million, a 260% increase that exceeded estimates by 55.8%. United Therapeutics was the weakest performer against analyst estimates, with revenues of $781.5 million down 1.6% year on year and missing by 1.9%. Amgen reported $8.62 billion in revenues, up 5.8% and beating estimates by 1.4%, and BioMarin Pharmaceutical recorded $766.2 million, up 2.8% but lagging expectations by 1.3%. Since their latest earnings results, therapeutics stocks have seen share prices rise 14% on average.
StockStory Highlights Comfort Systems and BioMarin as Cash-Rich Picks, Flags DocuSign as a Sell
StockStory identifies two cash-heavy stocks with exciting potential and one to ignore. DocuSign, with a net cash position of $630.8 million representing 7.4% of its market cap, is flagged as a sell due to underwhelming annual recurring revenue growth of 8.5% and high competitive spending. Comfort Systems, holding $1.01 billion in net cash or 1.6% of market cap, is viewed bullishly thanks to 53.1% average backlog growth over two years and a 9.5 percentage point increase in free cash flow margin over five years. BioMarin Pharmaceutical, with $782.8 million in net cash equating to 7.3% of market cap, is favored for its 14.5% annual revenue growth over two years and projected 28.4% revenue increase. Comfort Systems trades at $1,857 per share and 45.7 times forward earnings, while BioMarin is at $56.05 per share and 10.2 times forward earnings.
StockStory names BioMarin and Boston Scientific as top healthcare picks, flags ICU Medical as a sell
StockStory has identified BioMarin Pharmaceutical and Boston Scientific as two healthcare stocks poised for long-term outperformance, while recommending investors avoid ICU Medical. BioMarin, which develops therapies for rare genetic disorders, posted 14.5% annual sales growth over the past two years and is projected to accelerate to 28.4% revenue growth in the next twelve months, with its free cash flow margin expanding by 5.6 percentage points over five years. Boston Scientific, a maker of minimally invasive medical devices, achieved 15.7% organic revenue growth and grew earnings per share at a 24.2% annual rate over five years, supported by a 9.1 percentage point increase in free cash flow margin. In contrast, ICU Medical saw sales decline 1.6% annually over two years, earnings per share growth of just 2.9% annually over five years trailing revenue gains, and a return on invested capital of only 0.9%, signaling weak profitability and investment challenges.
BioMarin Pharmaceutical Q1 revenue misses estimates but full-year guidance raised
BioMarin Pharmaceutical reported first-quarter revenues of $766.2 million, up 2.8% year on year but falling short of analysts' expectations by 1.3%. The company achieved the highest full-year guidance raise among the 11 therapeutics stocks tracked, though it also posted a significant miss of analysts' EPS estimates. Moderna delivered the fastest revenue growth in the group, with revenues of $389 million surging 260% year on year and beating estimates by 55.8%. United Therapeutics was the weakest performer, with revenues of $781.5 million down 1.6% year on year and missing estimates by 1.9%. Overall, the therapeutics group beat consensus revenue estimates by 14.5% on average, and share prices have risen 7.6% on average since the latest earnings results.
BioMarin Reports Sustained Growth Benefits for VOXZOGO in Hypochondroplasia at ENDO 2026
BioMarin Pharmaceutical presented new clinical data at ENDO 2026 showing that three years of treatment with VOXZOGO led to sustained improvements in annualized growth velocity and height standard deviation scores for children with hypochondroplasia in an investigator-led Phase 2 extension study. The company plans to submit a supplemental New Drug Application to the FDA in the third quarter of 2026, building on recent positive Phase 3 results. BioMarin also shared Phase 1 data for BMN 333, an investigational long-acting C-type natriuretic peptide for achondroplasia, with a single-ascending dose study demonstrating sustained systemic exposure that supports a potential weekly dosing schedule. The treatment was well tolerated and significantly increased free CNP exposure compared to existing therapies, and enrollment is underway for a registration-enabling Phase 2/3 study.
USA