BioMarin

BioMarin Pharmaceutical Inc. is a biotechnology company that develops and commercializes therapies for rare genetic diseases. The company focuses on enzyme replacement therapies for mucopolysaccharidoses and other lysosomal storage disorders, as well as metabolic and other genetic conditions. BioMarin conducts research and development across its own programs and through collaborations, with a global commercial presence.

Notable approved therapies include Kuvan (sapropterin dihydrochloride) for phenylketonuria; Aldurazyme (laronidase) for mucopolysaccharidosis type I; Naglazyme (iduronidase) for mucopolysaccharidosis type VI; Vimizim (elosulfase alfa) for mucopolysaccharidosis type IVA; Brineura (cerliponase alfa) for CLN2 Batten disease; and Palynziq (pegvaliase-pqpz) for adults with phenylketonuria. These products have received regulatory approvals from the U.S. FDA and other international authorities.

BioMarin is headquartered in San Rafael, California. It is a publicly traded company listed on the NASDAQ stock market under the ticker symbol BMRN and maintains manufacturing operations and a global distribution network to support its portfolio and patient access.

The company pursues a strategy that combines commercialization of approved therapies with ongoing development programs aimed at expanding its rare-disease portfolio, including gene therapy and next-generation enzyme replacement approaches. This focus positions BioMarin as a leading entity in the specialty biotechnology sector serving underserved patient populations.