BioMarin Pharmaceutical Inc. discovers, develops, and commercializes innovative biopharmaceuticals for rare diseases driven by genetic mutations. The company's portfolio is anchored by its enzyme replacement therapies, which include treatments for various mucopolysaccharidosis (MPS) disorders such as Vimizim, Naglazyme, and Aldurazyme. Beyond ERTs, BioMarin's commercial products include Voxzogo for achondroplasia, Palynziq and Kuvan for phenylketonuria (PKU), Brineura for Batten disease, and the gene therapy Roctavian for severe hemophilia A. The company operates globally, with a commercial presence in approximately 80 countries across the Americas, Europe, the Middle East, and Asia-Pacific. BioMarin's business model relies on specialized research, targeted clinical development, and navigating complex regulatory pathways for orphan drugs.
