Group Petitions FDA To Require REMS If It Approves Hemophilia Gene Therapies

FDA should require a risk evaluation and mitigation strategy (REMS) to ensure the safety of two gene therapy treatments if it approves them for hemophilia, the National Hemophilia Foundation demands in a citizen petition filed with the agency. The two potential treatments are valoctocogene roxaparvovec, an investigational gene therapy for the treatment of severe hemophilia A made by BioMarin, and etranacogene dezaparvovec, an investigational gene therapy for hemophilia B made by CSL Behring. “We must ensure that the FDA implements...