FDA staff told advisers that Capricor’s deramiocel data do not adequately demonstrate the treatment works in Duchenne muscular dystrophy ahead of a Cellular, Tissue and Gene Therapies advisory committee meeting. The agency’s briefing documents argue the pivotal study’s design and analysis did not meet objectives for identifying efficacy. Capricor said previously that the Phase 3 trial met primary and secondary endpoints, but the FDA’s critique raises the risk of further scrutiny on how efficacy endpoints were interpreted and validated. The company is preparing for the July 29 discussion as it tries to navigate a path to potential approval for deramiocel in a space where regulators often require clear, reproducible efficacy signals.