BioMarin Pharmaceutical Inc. Company Profile
BioMarin Pharmaceutical Inc. develops and commercializes pharmaceuticals for serious diseases and medical conditions in the United States, Europe, Latin America, and internationally. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; Kuvan, a proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU), an inherited metabolic disease; Aldurazyme used for the treatment of patients with mucopolysaccharidosis I, a genetic disease; and Firdapse, a form of 3,4-diaminopyridine used for the treatment of Lambert Myasthenic Syndrome, an autoimmune disease. The company also conducts clinical trials on several investigational product candidates for the treatment of various diseases, including Kyndrisa, an exon-51 skipping compound for the treatment of Duchenne muscular dystrophy (DMD); pegvaliase, an enzyme substitution therapy for the treatment of PKU; reveglucosidase alfa, an enzyme replacement therapy for Pompe disease; vosoritide, a peptide therapeutic for the treatment of achondroplasia; BMN 044, BMN 045, and BMN 053 for DMD treatment; cerliponase alfa for the treatment of late infantile neuronal ceroid lipofuscinosis; and BMN 270, an AAV VIII vector and Factor VIII gene therapy drug development candidate for the treatment of hemophilia A. In addition, it develops preclinical product candidates for genetic and other metabolic diseases, such as alpha-N-acetyglucosaminidase. The company serves specialty pharmacies and end-users, such as hospitals and foreign government agencies; and distributors and pharmaceutical wholesalers. BioMarin Pharmaceutical Inc. has a collaboration agreement with Genzyme Corporation. The company was founded in 1996 and is headquartered in San Rafael, California.