Capricor Duchenne Drug Faces FDA Panel Amid Data Dispute
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- FDA's Cellular, Tissue, and Gene Therapies Advisory Committee convened Wednesday to review deramiocel, Capricor Therapeutics' cell therapy for Duchenne muscular dystrophy.
- Capricor Therapeutics reported in December that its Phase 3 trial showed deramiocel preserved upper-arm function and staved off heart failure with statistical significance on primary and secondary endpoints, positioning the drug as one of the only options for teenagers and young men with Duchenne who have already lost ambulation.
- FDA reviewers, in a harshly critical analysis posted Monday, concluded deramiocel failed to separate from placebo on the upper-limb and cardiac function endpoints.
- The FDA also flagged that Capricor made numerous changes to the study's statistical analysis plan after the trial was completed, further muddying the outcome.
Why it matters: With almost no approved treatments available for Duchenne patients who have lost the ability to walk, the committee's vote carries outsized weight for that population — and the FDA's public attack on post-hoc statistical changes puts Capricor's trial credibility directly before the panel that will weigh it.
