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BioMarin Pharmaceutical Inc. specializes in the development and commercialization of therapeutic solutions for individuals grappling with severe and life-threatening rare diseases. Its array of market-ready products features Vimizim, an enzyme replacement therapy for mucopolysaccharidosis (MPS) IV type A; Naglazyme, a recombinant N-acetylgalactosamine 4-sulfatase addressing MPS VI; and Kuvan, a proprietary oral synthetic 6R-BH4 compound used to manage phenylketonuria (PKU), an inherited metabolic disorder. The commercial portfolio also includes Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme delivered via subcutaneous injection to lower blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for ceroid lipofuscinosis type 2 (a form of Batten disease); Voxzogo, a daily injectable c-type natriuretic peptide analog for achondroplasia; and Aldurazyme, a purified protein mirroring the natural human enzyme alpha-L-iduronidase. The company further maintains a robust pipeline of investigational treatments. This includes valoctocogene roxaparvovec, an adeno-associated virus vector currently in Phase III clinical trials for severe hemophilia A; BMN 307, an AAV5-mediated gene therapy undergoing Phase 1/2 studies aimed at normalizing blood Phe levels in PKU patients; and BMN 255, which is in Phase 1/2 development for primary hyperoxaluria. BioMarin distributes its offerings to a diverse clientele, including specialty pharmacies, hospitals, non-U.S. government entities, distributors, and pharmaceutical wholesalers across the United States, Europe, Latin America, and other international territories. Strategic alliances for licensing and collaboration exist with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. BioMarin Pharmaceutical Inc. was established in 1996 and is based in San Rafael, California.
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