Capricor’s deramiocel was voted down by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee, with the panel concluding the available evidence for cardiomyopathy benefit in Duchenne muscular dystrophy was “very fragile.” The 9-3 vote capped a contentious meeting focused on the Phase 3 HOPE-3 trial’s secondary cardiac endpoint and the company’s choice of statistical analysis plan versions. FDA reviewers and external panelists disputed how the HOPE-3 results should be analyzed and whether the study demonstrated substantial evidence of effectiveness. Panelists cited concerns with the statistical framing and questioned whether the data sufficiently separated deramiocel from placebo. Capricor CEO Linda Marbán argued the agency considered a draft SAP that was not intended to be part of the BLA application review. Outside experts nonetheless found the evidence inadequate given the unmet need in an advanced DMD population. The outcome adds pressure to the FDA’s second review of deramiocel after a prior rejection, and it keeps the program’s path to approval uncertain for a therapy positioned for patients who have lost the ability to walk.