BioMarin Pharmaceutical US FDA Approved ROCTAVIAN
BioMarin Pharmaceutical (BMRN) announced that the U.S. FDA approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.
The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia . . .