BioMarin Pharmaceutical Inc. is a global biotechnology company that develops and commercializes therapies for rare genetic diseases and serious medical conditions. Founded in 1997 and headquartered in San Rafael, California, the company offers eight approved products including Aldurazyme (laronidase) for mucopolysaccharidosis type I; Naglazyme (galsulfase) for mucopolysaccharidosis type VI; Kuvan (sapropterin dihydrochloride) for phenylketonuria; Firdapse (amifampridine) for Lambert-Eaton myasthenic syndrome; Brineura (cerliponase alfa) for CLN2 disease; Vimizim (elosulfase alfa) for mucopolysaccharidosis type IVA; Palynziq (pegvaliase-pqpz) for phenylketonuria; Voxzogo (vosoritide) for achondroplasia; and Roctavian (valoctocogene roxaparvovec) gene therapy for severe hemophilia A. BioMarin operates in more than 70 countries across the Americas, Europe, Asia, and the Middle East, with manufacturing facilities in the United States and Ireland. In July 2025, the company completed its $270 million all-cash acquisition of Inozyme Pharma, adding INZ-701, a late-stage enzyme replacement therapy in development for ENPP1 deficiency and related rare conditions, to its pipeline; in 2024, the US FDA expanded approval of Brineura to children under three years old with CLN2 disease.