Posted by Michael Wonder on 08 Mar 2021
BioMarin announces FDA regenerative medicine advanced therapy designation granted to valoctocogene roxaparvovec, investigational gene therapy for haemophilia A
8 March 2021 - RMAT designation granted by FDA during bleeding disorders awareness month.
BioMarin Pharmaceutical today announced that the U.S. FDA granted regenerative medicine advanced therapy designation to valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe haemophilia A.
