BioMarin Pharmaceutical Inc. is a global biotechnology company engaged in the development of genetic discovery into medicines that make an impact on the life of each patient. The Company has a portfolio of commercial therapies and a clinical and preclinical pipeline. Its commercial products include Vimizim (elosulfase alpha), Naglazyme (galsulfase), Palynziq (pegvaliase-pqpz), Brineura (cerliponase alfa), Aldurazyme (laronidase), Roctavian (valoctocogene roxaparvovec), Kuvan (sapropterin dihydrochloride), and Voxzogo (vosoritide). Vimizim is an enzyme replacement therapy for the treatment of MPS IVA, a lysosomal storage disorder. The Roctavian is an adeno-associated virus (AAV5) vector gene therapy designed to restore factor VIII plasma concentrations in patients with severe hemophilia A. Its Galafold (migalastat) is the first oral treatment for Fabry disease, and Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat), is a two-component therapy for Pompe disease.