BMRn

BioMarin Pharmaceutical Inc., commonly referred to by its stock ticker BMRN, is an American biotechnology company that develops and commercializes therapies for rare genetic diseases. Based in San Rafael, California, it focuses on treatments for metabolic and genetic disorders and trades on the Nasdaq Stock Market under the ticker BMRN.

The company markets several approved therapies, including Naglazyme (galsulfase) for Morquio A syndrome (MPS VI); Kuvan (sapropterin dihydrochloride) as a pharmacologic chaperone for phenylketonuria; Vimizim (elosulfase alfa) for Morquio A syndrome (MPS IVA); Palynziq (pegvaliase) for adults with phenylketonuria who cannot maintain metabolic control; and Brineura (cerliponase alfa) for CLN2 disease, a form of neuronal ceroid lipofuscinosis.

In addition to these approved drugs, BioMarin develops gene therapies and other investigational programs, including Roctavian (valoctocogene roxaparvovec) for hemophilia A, which is in development.

The company pursues a strategy of developing therapies for rare genetic disorders with significant unmet medical need and markets products globally through its commercial and distribution network. BioMarin’s portfolio emphasizes enzyme replacement and precision pharmacology approaches intended to address diseases with limited approved treatment options.