Profile: Biomarin Pharmaceutical Inc (BMRN.OQ)
24 May 2019
BioMarin Pharmaceutical Inc., incorporated on October 25, 1996, is a biotechnology company. The Company develops and commercializes pharmaceuticals for various diseases and medical conditions. As of December 31, 2016, the Company's therapy portfolio consisted of five products, and multiple clinical and pre-clinical product candidates. Its commercial products include Aldurazyme (laronidase) for Mucopolysaccharidosis I (MPS I), Firdapse (amifampridine phosphate) for Lambert Eaton Myasthenic Syndrome (LEMS), Kuvan (sapropterin dihydrochloride) for phenylketonuria (PKU), Naglazyme (galsulfase) for Mucopolysaccharidosis VI (MPS VI) and Vimizim (elosulfase alpha) for Mucopolysaccharidosis IV Type A (MPS IV A).
The Company is conducting clinical trials on various product candidates for the treatment of various diseases. Its clinical product candidates include Brineura for the treatment of late infantile neuronal ceroid lipofuscinosis (CLN2); pegvaliase, an enzyme substitution therapy for the treatment of phenylketonuria (PKU); vosoritide, a peptide therapeutic for the treatment of achondroplasia; BMN 270, an AAV VIII vector and Factor VIII gene therapy drug development candidate for the treatment of hemophilia A, and BMN 250, a fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2) for the treatment of Sanfilippo B syndrome, or mucopolysaccharidosis type IIIB (MPS IIIB).
Aldurazyme is approved for marketing in the United States, the European Union (EU) and other international markets for patients with MPS I, a genetic disease that is caused by the deficiency of alpha-L-iduronidase. Kuvan is a synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for phenylalanine hydroxylase (PAH), indicated for patients with PKU, an inherited metabolic disease. Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) indicated for patients with MPS VI, a genetic disease that is caused by the deficiency of arylsulfatase B. Vimizim is an enzyme replacement therapy for the treatment of MPS IV A, a lysosomal storage disorder.
Product Candidates in Clinical Development
Brineura is a recombinant human tripeptidyl peptidase 1 in development for the treatment of patients with CLN2, a form of Batten disease. Pegvaliase is an investigational enzyme substitution therapy that it is developing as a subcutaneous injection for the treatment of PKU. Vosoritide is a peptide therapeutic in development for the treatment of achondroplasia. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. BMN 250 is an enzyme replacement therapy using a fusion NAGLU with a peptide derived from IGF2 for the treatment of MPS IIIB.
The Company competes with Spark Therapeutics, Inc., Alexion Pharmaceuticals Inc., Abeona Therapeutics Inc., Hoffmann-La Roche AG and Alnylam Pharmaceuticals, Inc.
Biomarin Pharmaceutical Inc
105 Digital Dr
NOVATO CA 94949-8703