Biomarin Pharmaceutical Inc. (BM8)

Overview

BioMarin Pharmaceutical Inc. is a distinguished entity focused on the development and commercialization of innovative therapies for individuals facing serious and life-threatening rare diseases and medical conditions. Since its establishment in 1996, the company has dedicated its efforts towards providing groundbreaking treatments for patients with limited therapeutic options. Headquartered in San Rafael, California, BioMarin operates on a global scale, serving specialty pharmacies, hospitals, and non-U.S. government agencies, in addition to engaging with distributors and pharmaceutical wholesalers across the United States, Europe, Latin America, and other international territories. Through its strategic license and collaboration agreements with notable entities like Sarepta Therapeutics, Ares Trading S.A., and Catalyst Pharmaceutical Partners, Inc., BioMarin seeks to expand its impact and reach within the rare disease community.

Products and Services

• Vimizim: An enzyme replacement therapy aimed at treating mucopolysaccharidosis (MPS) IV type A, a rare lysosomal storage disorder. Vimizim provides critical enzyme supplementation to address the underlying cause of the condition.
• Naglazyme: A recombinant form of N-acetylgalactosamine 4-sulfatase designed for patients with MPS VI. This product exemplifies BioMarin's commitment to addressing unmet medical needs within the lysosomal storage disorder community.
• Kuvan: A proprietary synthetic oral form of 6R-BH4, Kuvan stands as a cornerstone therapy for individuals with phenylketonuria (PKU), a genetic metabolic disease. It aids by modulating the phenylalanine hydroxylase enzyme, thereby managing phenylalanine levels in the body.
• Palynziq: This product is a PEGylated recombinant phenylalanine ammonia lyase enzyme administered through subcutaneous injection. It is aimed at reducing blood phenylalanine concentrations in patients, showcasing BioMarin's innovation in enzyme replacement therapies.
• Brineura: A recombinant human tripeptidyl peptidase 1 treatment for patients suffering from ceroid lipofuscinosis type 2, a form of Batten disease. Brineura underscores BioMarin's dedication to tackling neurodegenerative disorders.
• Voxzogo: A once-daily injection analog of c-type natriuretic peptide intended for achondroplasia patients. Voxzogo is a pioneering treatment aimed at addressing the root cause of dwarfism by promoting bone growth.
• Aldurazyme: Designed to be identical to the naturally occurring human enzyme alpha-L-iduronidase, Aldurazyme is a purified protein for MPS I patients. It represents a fundamental approach to treating lysosomal storage disorders by supplementing missing or deficient enzymes.
• Roctavian: Currently in development, Roctavian utilizes an adeno associated virus vector for patients with severe hemophilia A. This gene therapy represents BioMarin's cutting-edge research efforts to provide long-term solutions for hereditary blood disorders.