Biomarin Pharmaceutical

BioMarin Pharmaceutical, Inc., a biotechnology company, develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Aldurazyme that is used for the treatment of patients with mucopolysaccharidosis I, a genetic disease; Brineura for the treatment of late infantile neuronal ceroid lipofuscinosis type 2, a form of Batten disease; and Firdapse for Lambert Eaton Myasthenic Syndrome. The company's commercial products also comprise Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria, an inherited metabolic disease; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with mucopolysaccharidosis VI; and Vimizim, an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder. Its clinical and pre-clinical product pipeline includes pegvaliase, an enzyme substitution therapy for the treatment of phenylketonuria; vosoritide, a peptide therapeutic for the treatment of achondroplasia, a form of disproportionate short stature in humans; valoctocogene roxaparvovec, an AAV5 vector and factor VIII gene therapy drug development candidate for the treatment of severe hemophilia A; BMN 250, an investigational enzyme replacement therapy for the treatment of Sanfilippo Syndrome Type B or mucopolysaccharidosis type IIIB; and BMN 290, a selective chromatin modulation therapy for the treatment of Friedreich's ataxia. The company serves specialty pharmacies; and end-users, such as hospitals and foreign government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has collaboration agreements with Sarepta Therapeutics and Genzyme Corporation; and a license agreement with Asubio Pharma Co., Ltd. The company was founded in 1996 and is headquartered in San Rafael, California.