What FDA’s request for a new endpoint means for BioMarin’s hemophilia gene therapy

FDA has moved the goal post for BioMarin’s Roctavian, but the candidate could still be the first gene therapy to win approval for hemophilia A.

Shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) tumbled $41.83 (35%) to $76.71 Wednesday, losing $7.6 billion in market value, after FDA issued a complete response letter for Roctavian valoctocogene roxaparvovec. The company plans to meet with FDA in the coming weeks to align on next steps for the adeno-associated virus serotype 5 (AAV5) vector delivering Factor VIII. The therapy, which was evaluated under Priority Review, has breakthrough therapy and Orphan Drug designation. ...