BioMarin Wins FDA Approval for First Gene Therapy for Hemophilia A

The FDA has approved BioMarin Pharmaceutical’s valoctocogene roxaparvovec-rvox, to be marketed as Roctavian in the US, for the treatment of adult patients with severe hemophilia A. Roctavian, a one-time single-dose infusion, is authorized for use only in adults without antibodies against the adeno-associated virus serotype 5, as determined by an FDA-approved test.