Sarepta’s Duchenne Gene Therapy Does Not Have ‘Unambiguous Evidence,’ FDA Staff Says
Clinical studies to date “do not provide unambiguous evidence” for SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for ambulatory Duchenne muscular dystrophy patients, FDA reviewers said in briefing documents released ahead of an advisory committee meeting. The agency also raised safety concerns about the possibility of administering an ineffective gene therapy. Duchenne muscular dystrophy is […]