BioMarin Rolls Up Sleeves To Prepare For Roctavian’s EU Debut

With the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) positive opinion toward the gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A, BioMarin Pharmaceutical Inc. is now busy working to secure market access across Europe and estimates it could capture more than a third of eligible patients. The CHMP opinion makes European Commission approval likely, but it also drew cautious optimism regarding US Food and Drug Administration approval.