BioMarin Pharmaceutical Inc. Description
BioMarin Pharmaceutical Inc. develops and commercializes biopharmaceuticals for serious diseases and medical conditions in the United States, Europe, Latin America, and internationally. The company's commercial products include Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase enzyme used for the treatment of mucopolysaccharidosis VI; Kuvan, a proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU), a metabolic disease; Aldurazyme used for the treatment of mucopolysaccharidosis I, a genetic disease; and Firdapse used to treat Lambert Eaton Myasthenic Syndrome, an autoimmune disease. It also develops Vimizim, an enzyme replacement therapy that is in phase III clinical stage for the treatment of MPS IVA, a lysosomal storage disorder; PEG-PAL, an enzyme substitution therapy that is under Phase II clinical trial to treat PKU; BMN-701, an enzyme replacement therapy, which is under Phase I/II clinical trials for Pompe disease, a glycogen storage disorder; BMN-673, a clinical Phase I/II trial product for the treatment of cancer; and BMN-111, a peptide therapeutic that is under Phase I clinical trial for the treatment of achondroplasia. The company sells its Naglazyme, Kuvan, and Firdapse products to specialty pharmacies and end-users, such as hospitals and foreign government agencies, which act as retailers; and Naglazyme products to distributors and pharmaceutical wholesalers. It has a collaboration agreement with Genzyme Corporation for the manufacture of Aldurazyme; and an agreement with Merck Serono S.A. for the further development and commercialization of BH4 in Kuvan for PKU and for other indications, and PEG-PAL. BioMarin Pharmaceutical Inc. was founded in 1996 and is headquartered in San Rafael, California.
Classification
Sector:
Healthcare
Industry:
Biotechnology
Keywords:
Medicine
Biopharmaceutical
Cancer
Autoimmune Disease
Treatment Of Cancer
Drug Discovery
Rare Diseases
Virotherapy
Metabolic Disease
Genetic Diseases
Genzyme
Inborn Errors Of Metabolism
Enzyme Replacement Therapy
Lysosomal Storage Disorder
Pompe Disease
Mucopolysaccharidosis
Achondroplasia
Glycogen Storage Disorder
Iduronidase
Intellectual Disability
Lambert Eaton Myasthenic
Lambert Eaton Myasthenic Syndrome
Pharmaceutical Wholesalers
Phenylketonuria
Pku