BMRN - BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc. is a biotechnology company that focuses on the development
and commercialization of therapies for rare diseases and medical conditions. The company's
commercial products include Vimizim, Naglazyme, Kuvan, Palynziq, Brineura, Voxzogo, and Aldurazyme.
Vimizim is an enzyme replacement therapy for the treatment of mucopolysaccharidosis IV type A, while
Naglazyme is a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with
mucopolysaccharidosis VI. Kuvan is a synthetic oral form of 6R-BH4 that is used to treat
patients with phenylketonuria, an inherited metabolic disease. Palynziq is a PEGylated
recombinant phenylalanine ammonia lyase enzyme used to reduce blood Phe concentrations,
while Brineura is a recombinant human tripeptidyl peptidase 1 for the treatment of ceroid
lipofuscinosis type 2, a form of Batten disease. Voxzogo is a once-daily injection analog
of c-type natriuretic peptide for the treatment of achondroplasia, and Aldurazyme is a
purified protein designed to be identical to a naturally occurring form of the human
enzyme alpha-L-iduronidase.
In addition, BioMarin is developing several other products, including valoctocogene roxaparvovec, an adeno-associated virus vector in Phase III clinical trials for the treatment of severe hemophilia A; BMN 307, an AAV5-mediated gene therapy in Phase 1/2 clinical trials to normalize blood Phe concentration levels in patients with PKU; and BMN 255, which is in Phase 1/2 clinical trials for treating primary hyperoxaluria. BioMarin Pharmaceutical Inc. serves specialty pharmacies, hospitals, non-U.S. government agencies, distributors, and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. The company has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. BioMarin Pharmaceutical Inc. was founded in 1996 and is headquartered in San Rafael,
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