BioMarin Pharmaceutical, Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. The company is headquartered in San Rafael California, California and currently employs 3,040 full-time employees. The firm has eight 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 key clinical programs include Vosoritide, BMN 333, BMN 390, BMN 351, BMN 349, and INZ-701. Its late-stage enzyme replacement therapy, INZ-701, is being developed for the treatment of ENPP1 deficiency.
