BioMarin Pharmaceutical, Inc.
| Nasdaq: BMRN
BioMarin Pharmaceutical, Inc. develops and commercializes innovative pharmaceuticals for serious diseases and medical conditions. The company operates its business through one segment, the biopharmaceutical development and commercialization segment. Its company's product portfolio is comprised of five approved products and multiple investigational product candidates which include Naglazyme, Kuvan, Aldurazyme, Firdapse and VIMIZIM. Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase indicated for patients with ucopolysaccharidosis VI. MPS VI is a debilitating life-threatening genetic disease for which no other drug treatment currently exists and is caused by the deficiency of arylsulfatase B, an enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans, or GAGs. Kuvan is a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for phenylalanine hydroxylase, indicated for patients with PKU, which is an inherited metabolic disease that affects patients under the age of 40 in the developed world. Aldurazyme has been approved for marketing in the U.S., EU and other countries for patients with mucopolysaccharidosis. Firdapse is a form of 3, 4-diaminopyridine for the treatment of Lambert Myasthenic Syndrome. And VIMIZIM is an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder. The company was founded on March 21, 1997 and is headquartered in Novato, CA.