Discovers, develops, and commercializes innovative pharmaceuticals for serious diseases and medical conditions
BioMarin Pharmaceutical, Inc. develops and commercializes innovative pharmaceuticals for serious diseases and medical conditions.
The company's product portfolio is comprised of four approved products and multiple investigational product candidates which include Naglazyme, Kuvan, Aldurazyme and Firdapse.
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 I (MPS I). MPS I is a progressive and debilitating life-threatening genetic disease, for which no other drug treatment currently exists, that is caused by the deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of GAGs.
The company is conducting clinical trials on several investigational product candidates for the treatment of various diseases, including GALNS, an enzyme replacement therapy for the treatment of Mucopolysaccharidosis Type IV; PEG-PAL, an enzyme substitution therapy for the treatment of phenylketonuria; BMN-701, an enzyme replacement therapy for Pompe disease, a glycogen storage disorder; and BMN-673, an orally available poly polymerase, inhibitor for the treatment of patients with cancer.
It is also conducting preclinical development of several other enzyme product candidates for genetic and other metabolic diseases, including BMN-111, a peptide therapeutic for the treatment of achondroplasia.
The company was founded on March 21, 1997 and is headquartered in Novato, CA.