"FDA's Peter Marks Leans Toward Full Approval for Sarepta's Duchenne Gene Therapy"

Peter Marks, the FDA's top gene therapy regulator, appears open to granting some form of full approval to Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy, Elevidys, despite the failure of a confirmatory study in October. Marks suggested that the FDA has approved products based on aggregate scale performance, indicating a potential willingness to overlook certain imperfections in the data.