FDA Staff Finds Duchenne Muscular Dystrophy Treatment Lacks Sufficient Evidence

FDA briefing documents indicate that Capricor Therapeutics' cell therapy, deramiocel, for Duchenne muscular dystrophy, did not provide "substantial evidence of effectiveness." The agency noted reliance on a single pivotal trial, typically requiring at least two for approval. This assessment led to a significant drop in Capricor's stock value and raises concerns for patients awaiting new treatments.