Description

BioMarin Pharmaceutical Inc. is dedicated to discovering, developing, and marketing treatments for individuals suffering from serious and life-threatening rare diseases and medical conditions. Its current product portfolio features several key therapies: Vimizim, an enzyme replacement addressing mucopolysaccharidosis (MPS) IV type A; Naglazyme, a recombinant N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary oral formulation of 6R-BH4 used in the management of phenylketonuria (PKU). Other commercial offerings include Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme administered via subcutaneous injection to reduce blood phenylalanine concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for treating ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a daily injectable analog of c-type natriuretic peptide for achondroplasia; and Aldurazyme, a purified protein designed to replicate the human enzyme alpha-L-iduronidase. The company also maintains a robust development pipeline, which includes Roctavian, an adeno-associated virus vector therapy for severe hemophilia A. Furthermore, BMN 331, an AAV5-mediated gene therapy, is progressing through Phase 1/2 clinical trials for Hereditary Angioedema (HAE), and BMN 255 is undergoing similar Phase 1/2 studies for primary hyperoxaluria. BioMarin serves a diverse clientele, including specialty pharmacies, hospitals, non-U.S. government health agencies, distributors, and pharmaceutical wholesalers across the United States, Europe, Latin America, and globally. Incorporated in 1996 and based in San Rafael, California, the company also engages in license and collaboration agreements with entities like Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd.