Capricor Therapeutics received a Complete Response Letter from the FDA rejecting the Biologics License Application for deramiocel, a cell therapy for cardiomyopathy in Duchenne muscular dystrophy patients. The FDA cited inadequate substantial evidence of efficacy and outstanding chemistry, manufacturing, and controls issues. Capricor expressed surprise and is awaiting additional Phase III data to support resubmission. The rejection reflects a possible shift in FDA’s regulatory flexibility policies under new leadership, contrasting with prior openness toward novel cell and gene therapies for rare diseases.