USFDAJul 30, 2026, 09:32 AM
Capricor's Deramiocel Fails FDA Advisory Committee Vote (3 for, 9 against)
AI Summary
Capricor Therapeutics announced that the FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted against the effectiveness of Deramiocel for treating cardiomyopathy in Duchenne muscular dystrophy (DMD) patients, with a vote of 3 for, 9 against, and 0 abstain. While the vote is non-binding and addressed a narrower indication, the committee's feedback on upper limb function was directionally supportive. Capricor remains committed to Deramiocel, expressing confidence in its HOPE-3 data, and continues to work with the FDA towards a potential approval by the August 22, 2026 PDUFA target action date.
Key Highlights
- FDA Advisory Committee voted 3 for, 9 against, 0 abstain on Deramiocel's effectiveness for cardiomyopathy in DMD.
- The Committee's vote is non-binding and addressed a narrower indication than proposed.
- Feedback on upper limb function from the Phase 3 HOPE-3 trial was directionally supportive.
- Capricor Therapeutics remains committed to Deramiocel and confident in the HOPE-3 data.
- The company is working with the FDA toward a potential approval ahead of the August 22, 2026 PDUFA date.
- Deramiocel has Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT), and Rare Pediatric Disease Designations.
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