The FDA raised new questions about Capricor’s deramiocel effectiveness in Duchenne muscular dystrophy, according to briefing materials ahead of a Cellular, Tissue and Gene Therapies Advisory Committee meeting. Capricor said its Phase 3 program met primary and secondary endpoints, while the agency’s review team reported it did not meet the objectives. Capricor’s shares fell sharply as investors digested the FDA tone, and the company issued a response tying the agency concerns to the use of an incomplete, unsigned statistical analysis plan draft rather than the final SAP used for the company’s positive analysis. The advisory committee meeting is now framed as a key inflection point for whether the FDA’s concerns can be resolved for a potential BLA path, with stakeholders watching for how the agency and the company reconcile the statistical record.