The FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9–3 against Capricor's cell therapy, deramiocel, for treating cardiomyopathy in Duchenne muscular dystrophy.
The disagreement centers on statistical analysis methods, including changes Capricor made to the statistical analysis plan after the HOPE-3 trial ended, and the FDA's use of an earlier, different SAP version that showed no significant effect.
The vote is nonbinding, but approval is unlikely, with a PDUFA decision expected August 22, 2026.
Sources:
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