BMRN 📈 Biomarin Pharmaceutical - Overview

BioMarin Pharmaceutical Inc. is a biotechnology company that focuses on developing and commercializing innovative therapies for people with rare and life-threatening diseases. The company's product portfolio includes a range of enzyme replacement therapies, such as Vimizim, which is used to treat mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder that affects the breakdown of sugar molecules in the body. Another key product is Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase, which is used to treat patients with MPS VI, a similar lysosomal storage disorder. Additionally, the company offers Kuvan, a synthetic oral form of 6R-BH4, which is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease that affects the body's ability to break down the amino acid phenylalanine.

The company's commercial products also include Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations in patients with PKU. Brineura, a recombinant human tripeptidyl peptidase 1, is used to treat patients with ceroid lipofuscinosis type 2, a form of Batten disease that affects the nervous system. Voxzogo, a once-daily injection analog of c-type natriuretic peptide, is used to treat achondroplasia, a genetic disorder that affects bone growth. Furthermore, the company offers Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase, which is used to treat patients with MPS I, a lysosomal storage disorder. These products are designed to address the significant unmet medical needs of patients with rare and serious diseases.

BioMarin Pharmaceutical Inc. is also involved in the development of new therapies, including Roctavian, an adeno-associated virus vector, which is being developed for the treatment of patients with severe hemophilia A, a genetic disorder that affects the blood's ability to clot. The company serves a range of customers, including specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. Through its license and collaboration agreements with companies such as Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and CAMP4 Therapeutics Corporation, BioMarin Pharmaceutical Inc. is able to leverage its expertise and resources to develop and commercialize innovative therapies for patients with rare and serious diseases.

As a company, BioMarin Pharmaceutical Inc. has a strong foundation in research and development, with a focus on creating innovative therapies that address significant unmet medical needs. With its headquarters in San Rafael, California, the company is well-positioned to collaborate with other biotechnology and pharmaceutical companies, as well as academic and research institutions, to advance the development of new therapies. Through its website, https://www.biomarin.com, the company provides information on its products, research and development activities, and corporate governance. With its common stock listed on the NASDAQ exchange under the ticker symbol BMRN, and its ISIN code US09061G1013, BioMarin Pharmaceutical Inc. is a publicly traded company that is subject to the reporting requirements of the Securities and Exchange Commission.