BioMarin Banks on More Robust Data in Hemophilia A Resubmission

Two years after receiving a Complete Response Letter for its hemophilia gene therapy, BioMarin is heading back to the FDA with stronger and more robust data for potential approval.

California-based BioMarin resubmitted a Biologics License Application for valoctocogene roxaparvovec, a potential one-time gene therapy treatment for severe hemophilia A. If approved, it would mark the first gene therapy in the United States for the treatment of this disease. The gene therapy was authorized for use in Europe in August under the brand name Roctavian.