BioMarin Pharmaceutical Inc. develops and commercializes biopharmaceuticals for serious diseases and medical conditions. Its product portfolio comprises approved products and multiple investigational product candidates. The company's approved products include Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase enzyme used to treat genetic disease called mucopolysaccharidosis (MPS)VI; Kuvan tablet, a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for phenylalanine hydroxylase indicated for patients with Phenylketonuria (PKU); Aldurazyme, which is used to treat mucopolysaccharidosis I caused by the deficiency of a lysosomal enzyme called alpha-L-iduronidase; and Firdapse, an orphan drug for the treatment of lambert eaton myasthenic syndrome. Its other product candidates include PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is under Phase II clinical development for the treatment of PKU; GALNS (N-acetylgalactosamine 6-sulfatase), which is under in Phase I/II clinical development for the treatment of MPS IVA; and BMN 195, a Phase I clinical development product for the treatment of duchenne muscular dystrophy. The company is also conducting preclinical development of various other enzyme product candidates for genetic and other metabolic diseases, including BMN-185, an IgA protease for IgA nephropathy; and BMN-103, a glucosidase for pompe disease. In addition, BioMarin holds rights to receive royalties related to Orapred and Orapred ODT as a result of sublicense of North American rights. The company sells its products to specialty pharmacies, hospitals acting as retailers, pharmaceutical wholesalers, and distributors throughout the world. BioMarin has a strategic alliance with Merck Serono S.A. and collaboration with Genzyme Corporation
