Capricor’s Deramiocel faced another major setback as the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee recommended against approval following its resubmitted biologics license application. In a 9–3 vote, the panel concluded that evidence from the phase 3 HOPE-3 trial (NCT05126758) did not provide substantial evidence of effectiveness for cardiomyopathy in Duchenne muscular dystrophy. The advisory committee decision is expected to influence the FDA’s review as the agency targets an Aug. 22 decision date under the Prescription Drug User Fee Act. FDA staff previously highlighted concerns about endpoint performance, a higher hypersensitivity rate in Deramiocel-treated patients (42% vs. 15% for placebo), and the lack of an updated statistical analysis plan provided before the February resubmission. Deramiocel is an allogeneic cardiosphere-derived cell (CDC) therapy designed to modulate macrophages via secreted exosomes, aiming to shift them toward a healing phenotype. Capricor will now need to address the panel’s concerns ahead of the FDA ruling.