BioMarin Pharmaceutical Inc.
NASDAQ Global SelectCapital Structure
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in mil. unless spec.Company Description
- CEO
- Alexander Hardy
- Full Time Employees
- 3,221
- Sector
- Healthcare
- Industry
- Biotechnology
- Address
- 770 Lindaro Street San Rafael CA United States of America 94901
- IPO Date
- Jul 26, 1999
- CIK
- 0001048477
- Website
- biomarin.com
- Similar Companies
- Business
- BioMarin Pharmaceutical Inc. is a global biotechnology company specializing in the development and commercialization of innovative therapies for rare genetic diseases and serious medical conditions. Founded in 1997 and headquartered in San Rafael, California, BioMarin offers a portfolio of approved products including VOXZOGO for achondroplasia and hypochondroplasia; ROCTAVIAN for severe hemophilia A; BRINEURA for Batten disease; and ALDURAZYME for mucopolysaccharidosis I. The company’s product pipeline includes advanced investigational therapies such as BMN 351 for Duchenne muscular dystrophy, BMN 349 for alpha-1 antitrypsin deficiency-associated liver disease, and BMN 333 for multiple growth disorders. BioMarin employs multiple treatment modalities including enzyme replacement therapies, oligonucleotide therapeutics, and gene therapies. Recent major developments include BioMarin’s acquisition of Inozyme Pharma in mid-2025 for approximately $270 million, which expanded BioMarin’s portfolio with INZ-701, a late-stage enzyme replacement therapy in Phase 3 development targeting ENPP1 deficiency, a rare genetic disorder affecting blood vessels and connective tissues. Additionally, BioMarin advanced its pivotal Phase 3 clinical trial for VOXZOGO in hypochondroplasia with topline data expected in 2026 and a potential product launch in 2027. The company also strengthened its research capabilities through collaborations such as the partnership with Deep Genomics to apply artificial intelligence for oligonucleotide drug discovery. In 2023 and 2024, BioMarin focused its R&D strategy by prioritizing three key clinical assets and discontinuing four earlier-stage candidates, accelerating the pace of new drug filings and advancing pipeline productivity. BioMarin operates primarily in the rare disease biopharmaceutical sector with a focus on genetic and lysosomal storage disorders, serving patients across North America, Europe, and other global markets. The company integrates research, development, manufacturing, and commercialization functions, leveraging scientific expertise to bring first- or best-in-class genetic therapies to market. BioMarin’s efforts are supported by strategic alliances and a growing portfolio of proprietary products and clinical-stage candidates aimed at addressing high unmet medical needs in genetic diseases.
Company News
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BioMarin Announces Global Settlement with Ascendis Pharma A/S; Ascendis Will Pay Royalties to BioMarin on Yuviwel Sales in U.S., EU, Brazil and South Korea
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