FDA is signaling major concerns around Capricor Therapeutics’ Duchenne muscular dystrophy stem-cell therapy deramiocel after questioning whether Phase 3 objectives were met, according to the agency’s pre-hearing communication. Capricor had previously reported in December that deramiocel met both primary and secondary endpoints in a large randomized study, making the FDA’s stated nonconformance a key inflection point for next steps. For Duchenne programs, the dispute underscores how endpoint interpretation and post-hoc consistency will remain central as gene- and cell-therapy developers prepare for regulatory review and label-extension conversations.