BioMarin

BioMarin is a biopharmaceutical company that develops therapeutics for rare genetic diseases. The company focuses on conditions that affect small patient populations, most of whom are children.

BioMarin’s cGMP manufacturing facility is in Novato, California. In 2011 the company acquired a biologics manufacturing plant from Pfizer in Shanbally, Cork, Ireland. BioMarin also operates process development and pilot scale laboratories to evaluate investigational product candidates.

• Palynziq (pegvaliase-pqpz) Injection self-administered enzyme substitution therapy for PKU
• Kuvan (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution for PKU
• Brineura (cerliponase alfa) for CLN2 Disease
• Vimizim (elosulfase alfa) for Morquio A Syndrome
• Naglazyme (galsulfase) for MPS VI
• Firdapse (amifampridine phosphate) (only approved in the EU) for LEMS
• Aldurazyme (laronidase) for MPS I

• Vosoritide (BMN 111) is designed for the treatment of achondroplasia, the most common form of dwarfism
• Tralesinidase alfa (BMN 250) for MPS IIIB is an enzyme replacement therapy being developed to treat Sanfilippo B Syndrome

• Valoctogene roxaparvovec (valrox) is a gene therapy for hemophilia in clinical trials as of 2019
• BMN 307 for PKU, plans to file an application for beginning clinical trials for was reported in 2019.