Capricor Therapeutics received an FDA complete response letter (CRL) for deramiocel, its off-the-shelf cell therapy targeting cardiomyopathy in Duchenne muscular dystrophy (DMD). The regulator cited insufficient evidence of efficacy and outstanding chemistry, manufacturing, and controls (CMC) issues. The unexpected setback follows FDA leadership changes and delays the prioritized biologics license application, impacting expectations for DMD treatment options.