BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California, founded in 1997. The company's core business revolves around the development and commercialization of innovative therapies for individuals living with serious and life-threatening rare genetic diseases and medical conditions. Its mission is to translate genetic discoveries into new medicines that profoundly impact patients' lives.

BioMarin's product portfolio includes eight approved therapies, specializing in enzyme replacement therapies, gene therapies, and other advanced treatments. Key products address conditions such as achondroplasia, phenylketonuria (PKU), hemophilia, and various mucopolysaccharidosis (MPS) types. Notable marketed products include Kuvan, Naglazyme, Aldurazyme, Firdapse, Vimizim, Brineura, Palynziq, Roctavian, and Voxzogo.

In April 2026, BioMarin completed the acquisition of Amicus Therapeutics for approximately $4.8 billion, significantly expanding its commercial portfolio with treatments for Fabry disease (Galafold) and Pompe disease (Pombiliti + Opfolda), and securing U.S. rights to DMX-200 for focal segmental glomerulosclerosis (FSGS). Alexander Hardy serves as the President and CEO, and Ian T. Clark was appointed Chair of the Board in April 2026. The company maintains a strong market position as a global leader in rare disease biotechnology, with a focus on orphan drugs and a robust pipeline.