BioMarin Pharmaceutical Inc - Company Profile
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Business Description
BioMarin Pharmaceutical Inc (BioMarin) is a biotechnology company focused on translating genetic discoveries into medicines that address rare and difficult-to-treat genetic conditions. BioMarin's primary activities include drug discovery, development, and manufacturing, with a focus on genetic conditions such as achondroplasia, severe hemophilia A, and various lysosomal storage diseases.
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R&D Overview
BioMarin's research and development (R&D) initiatives focus on leveraging genetic insights to develop transformative medicines for a broad range of serious medical conditions. The company applies its expertise across multiple treatment modalities, including biologics, gene therapy, oligonucleotides, peptides, and small molecules. BioMarin's R&D program focuses on introducing new medicines built on genetic insights, with a commitment to addressing the root causes of genetic conditions with significant unmet medical needs. The R&D unit of the company is responsible for the research and discovery of new product candidates and supporting the development and registration efforts for potential new products. The unit plays a crucial role in managing the company's R&D activities, ensuring a deep understanding of the underlying mechanisms of disease and applying this knowledge to develop innovative treatments. As of December 2025, the company had 692 personnel working towards its R&D. In FY2025, the company spent US$921.9 million on its R&D activities, which as a percentage of revenue stood at 28.6% and grew 23.4% YoY. Its Phase III products include vosoritide to treat hypochondroplasia and BMN 401 to treat ENPP1 deficiency. Its Phase II products include vosoritide to treat idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency; BMN 351 to treat Duchenne Muscular Dystrophy (DMD); and BMN 401 to treat ABCC6 deficiency.
Product Categories
Overview
It is a highly purified protein, which is designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of GAGs. Used to treat patients with MPS I. Developed through collaboration with Sanofi. Marketed in the US, the EU, and other international markets.
Performance
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Overview
It is a recombinant human tripeptidyl peptidase 1 (TPP1) for the treatment of patients with CLN2, a form of Batten disease. Administered via intracerebroventricular (ICV) infusion and intended to be used in combination with a delivery device, such as an injector or other delivery system. Marketed in the US, the EU, and other international countries for children of all ages.
Performance
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Overview
It is a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for PAH, indicated for patients with PKU. Marketed in the US, the EU, and other international markets (excluding Japan). In certain international markets, it's also approved for the treatment of primary BH4 deficiency, a different disorder from PKU.
Performance
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Overview
It is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) to treat patients with MPS VI. Marketed in the US, the EU, and other international markets.
Performance
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Overview
It is a PEGylated recombinant phenylalanine (Phe) ammonia lyase enzyme, delivered through subcutaneous injection to reduce blood Phe concentrations. Offered to adult patients with Phenylketonuria (PKU) who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management in the US. In the EU, Australia, and Brazil offered to patients aged 16 and older who have inadequate blood Phe control (blood Phe concentrations greater than 600 micromol/L) despite prior management with available treatment options were offered.
Performance
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Overview
It is an adeno-associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Marketed in the US.
Performance
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Overview
Includes receipt of sales-based royalties. It also includes income from licenses of intellectual property and milestone payments.
Performance
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Overview
It is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. Marketed in the US, the European Union (EU), and other international markets.
Performance
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Overview
It is a once-daily injection analog of C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia. Majorly approved for marketing in the US and Japan to treat achondroplasia in children with open growth plates of all ages, in the EU to treat children with open growth plates aged four months and older, and in other markets, including Australia and Brazil, for patients in various age ranges.
Performance
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