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. Galafold (migalastat) is its oral treatment for Fabry disease, and Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat), is a two-component therapy for Pompe disease. Its portfolio also includes ALE1, an oral small molecule for the potential treatment of hypophosphatasia (HPP), a rare genetic bone disease caused by mutations in the ALPL gene.