FDA Advisory Panel Rejects Capricor's Deramiocel Cell Therapy for Duchenne Cardiomyopathy

On July 29, 2026, an FDA advisory panel voted 9-3 against Capricor Therapeutics' cell therapy, deramiocel, citing a lack of substantial evidence for treating Duchenne muscular dystrophy cardiomyopathy. Although Capricor defended its Phase 3 trial data, FDA reviewers argued statistical changes masked a lack of efficacy. A Lancet publication also showed the drug missed statistical significance on heart benefits. The FDA will make a final approval decision by August 22, 2026.

FDA advisory panel rejection

• ▪The Food and Drug Administration is expected to make a final decision on Capricor Therapeutics' revised application for deramiocel by August 22, 2026.
• ▪A Food and Drug Administration advisory panel voted 9-3 on July 29, 2026, that Capricor Therapeutics' deramiocel did not show substantial evidence of effectiveness.

Phase 3 trial data disputes

• ▪The Lancet publication revealed deramiocel missed statistical significance on purported heart benefits, prompting Capricor Therapeutics to issue a regulatory clarification.
• ▪Full results of Capricor Therapeutics' Phase 3 trial for deramiocel were published in The Lancet on July 29, 2026.

FDA regulatory review history

• ▪The Food and Drug Administration previously rejected Capricor Therapeutics' deramiocel in July 2025 before agreeing to review a revised submission.
• ▪Former Food and Drug Administration Commissioner Marty Makary and former deputy Vinay Prasad departed the agency prior to the July 2026 advisory panel meeting.

Statistical analysis methodology controversy

• ▪Capricor Therapeutics countered that Food and Drug Administration staff relied on an outdated analysis and defended its finalized pre-unblinding statistical plan.
• ▪Food and Drug Administration reviewers argued Capricor Therapeutics changed its statistical analysis plan to make deramiocel's Phase 3 results appear beneficial.

Panel expert opinions

• ▪Some advisory panel members noted that data regarding deramiocel's impact on upper limb function was more favorable than the cardiomyopathy data.
• ▪Food and Drug Administration advisory panel members characterized Capricor Therapeutics' Phase 3 trial results for deramiocel as fragile.

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