BMRN - Biomarin Pharmaceutical - Stock & Dividends

BioMarin Pharmaceutical Inc. is a biotech company that focuses on developing and commercializing innovative therapies for people living with rare and life-threatening genetic diseases. These diseases often have limited treatment options, and BioMarin's mission is to provide new hope for patients and their families.

The company has a portfolio of commercial products that address specific rare diseases. For instance, Vimizim is an enzyme replacement therapy for mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder that affects the body's ability to break down sugar molecules. Naglazyme, on the other hand, is a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI, another lysosomal storage disorder.

BioMarin's product lineup also includes Kuvan, a synthetic oral form of 6R-BH4 that helps treat patients with phenylketonuria (PKU), an inherited metabolic disease that affects the body's ability to process protein. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is another product that reduces blood Phe concentrations in PKU patients.

In addition, the company offers Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of ceroid lipofuscinosis type 2, a form of Batten disease, a rare and fatal neurodegenerative disorder. Voxzogo, a once-daily injection analog of c-type natriuretic peptide, is used to treat achondroplasia, a genetic disorder that affects bone growth. Aldurazyme, a purified protein, is designed to replace a naturally occurring form of the human enzyme alpha-L-iduronidase.

BioMarin is also developing Roctavian, an adeno-associated virus vector, for the treatment of severe hemophilia A, a genetic disorder that affects blood clotting. The company has partnerships with other biotech companies, such as Sarepta Therapeutics, Ares Trading S.A., and Catalyst Pharmaceutical Partners, Inc., to advance its research and development efforts.

BioMarin serves a global customer base, including specialty pharmacies, hospitals, and government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and other international markets. The company was founded in 1996 and is headquartered in San Rafael, California.