BioMarin

Company Overview

BioMarin Pharmaceutical Inc. is a global biotechnology company that specializes in developing and commercializing innovative therapies for rare and serious genetic diseases. Founded in 1997 and headquartered in San Rafael, California, BioMarin focuses on providing life-changing treatments for conditions that often have limited or no available treatments. The company's portfolio includes therapies for hemophilia, metabolic disorders, and rare genetic diseases, emphasizing enzyme replacement therapies, gene therapies, and small molecule treatments.

BioMarin is known for its powerful pipeline of drugs, including FDA-approved medicines such as Naglazyme for the treatment of mucopolysaccharidosis VI, Vimizim for Morquio A syndrome, and Kuvan for phenylketonuria. It is committed to integrating leading scientific research with its mission to solve unmet medical needs. Its strategic partnerships, coupled with increasingly significant research and development efforts, continue to take BioMarin to the future in rare diseases, working on improving patient outcomes and quality of life worldwide.

Financial Highlights (FY 2024)

Company's Business Segments

• Enzyme : VIMIZIM, NAGLAZYME, PALYNZIQ, BRINEURA, ALDURAZYME, Others
• VOXZOGO : C-type Natriuretic Peptide (CNP) for the treatment of achondroplasia
• KUVAN : KUVAN is a proprietary synthetic oral form of 6R-BH4 a naturally occurring enzyme co-factor for PAH
• ROCTAVIAN : ROCTAVIAN is an adeno associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A.