FDA reviewers raised new efficacy questions about Capricor’s Duchenne muscular dystrophy cell therapy, deramiocel, ahead of an advisory committee meeting. The agency said it could not confirm that the program met the trial’s objectives, contradicting the company’s earlier claims following its prior readout. Capricor also tied concerns to the FDA’s use of a different statistical analysis approach than the plan it said supported its positive results. Ahead of the adcom, the debate centers on the statistical analysis plan version and whether the company’s prespecified objectives were appropriately addressed. For the broader cell therapy space, the dispute highlights how analysis-plan differences can become decisive in late-stage evidence packages, even when safety and overall response messaging appears constructive.