Capricor’s Deramiocel has been rejected again by an FDA advisory committee, with members concluding that the evidence from the HOPE-3 phase 3 trial does not provide substantial effectiveness for the therapy in Duchenne muscular dystrophy (DMD) cardiomyopathy. At the FDA Cellular, Tissue, and Gene Therapies Advisory Committee meeting, the committee voted 9–3 against approval of Deramiocel. The panel also reviewed FDA staff concerns tied to endpoints, reported hypersensitivity events, and the company’s resubmitted biologics license application materials. Deramiocel is an allogeneic cardiosphere-derived cell therapy intended to exert immunomodulatory and anti-fibrotic effects through exosome secretion. Capricor is awaiting an FDA decision under a PDUFA target date of Aug. 22. The outcome increases the likelihood that the FDA will require additional evidence or analyses before moving forward in a setting where confirmatory effectiveness remains a central regulatory gate.