FDA Rejects Capricor Cell Therapy for Duchenne Cardiomyopathy

The U.S. Food and Drug Administration has issued a Complete Response Letter rejecting Capricor Therapeutics' Biologics License Application for Deramiocel, a cell therapy aimed at treating cardiomyopathy associated with Duchenne muscular dystrophy. The FDA cited insufficient evidence of effectiveness and requested additional clinical data, notably from Capricor's ongoing Phase 3 HOPE-3 trial, with topline results expected in the third quarter of 2025. Despite this setback, Deramiocel holds several regulatory designations, including Orphan Drug and Regenerative Medicine Advanced Therapy, which may facilitate future approval processes. Capricor plans to resubmit the application incorporating the new trial data and will request a Type A meeting with the FDA to discuss next steps. The FDA also noted outstanding Chemistry, Manufacturing, and Controls issues, most of which Capricor believes have already been addressed. This rejection delays the anticipated approval timeline after the application had previously received Priority Review status earlier in 2025.