FDA briefing documents again raised questions about the efficacy of Capricor’s Duchenne muscular dystrophy cell therapy deramiocel ahead of an advisory committee meeting. The FDA said it could not identify that the Phase 3 program met its stated objectives, contrasting with Capricor’s earlier claims that the therapy achieved primary and secondary endpoints in the randomized study. Capricor responded publicly, disputing the FDA’s critique of the statistical analysis approach and pointing to differences between analysis plans. The dispute is playing out as investors weigh another potential regulatory outcome after repeated scrutiny for cell therapies in DMD. For developers and stakeholders, the case highlights how trial design and statistical framing can be decisive in whether cell therapy efficacy is accepted by regulators.