BioMarin Pharmaceutical Inc. specializes in creating and bringing to market treatments for individuals suffering from severe and often fatal rare disorders and other critical medical conditions. The company currently markets several key pharmaceutical products. Among these are Vimizim, an enzyme replacement therapy targeting mucopolysaccharidosis (MPS) IV type A, a specific lysosomal storage disorder, and Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for individuals with MPS VI. Another notable offering is Kuvan, a unique synthetic oral version of 6R-BH4, prescribed for phenylketonuria (PKU), an inherited metabolic condition. Further expanding its product portfolio, BioMarin provides Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme administered via subcutaneous injection to lower blood Phe levels. Also available are Brineura, a recombinant human tripeptidyl peptidase 1 used in treating ceroid lipofuscinosis type 2, a variant of Batten disease; Voxzogo, a daily injectable c-type natriuretic peptide analog for achondroplasia; and Aldurazyme, a refined protein mirroring the naturally occurring human enzyme alpha-L-iduronidase. Beyond its established products, BioMarin has a robust development pipeline. This includes valoctocogene roxaparvovec, an adeno-associated virus vector undergoing Phase III clinical trials for severe hemophilia A. Additionally, BMN 307, an AAV5-mediated gene therapy, is in Phase 1/2 trials, aiming to normalize blood phenylalanine levels in PKU patients, while BMN 255 is also in Phase 1/2 trials for primary hyperoxaluria. Its clientele spans specialty pharmacies, hospitals, international government health bodies, and a network of distributors and pharmaceutical wholesalers. These services reach patients and healthcare providers across the United States, Europe, Latin America, and other global markets. BioMarin maintains strategic licensing and collaborative alliances with partners such as Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. Established in 1996, the company's corporate headquarters are located in San Rafael, California.
