Capricor Therapeutics faced a renewed setback for its Duchenne muscular dystrophy cardiomyopathy program as the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee recommended against approval of Deramiocel. The panel voted 9–3 that evidence from the phase 3 HOPE-3 trial did not provide “substantial evidence of effectiveness.” The advisory decision comes as Capricor works through a resubmitted BLA under a PDUFA target date of August 22. The panel’s recommendation is likely to shape how the FDA evaluates the application, even though the agency is not required to follow adcomm outcomes. FDA reviewers previously flagged concerns around endpoint achievement in HOPE-3 and the earlier HOPE-2 study, hypersensitivity rates (42% in Deramiocel patients versus 15% on placebo), and the company’s failure to submit an updated statistical analysis plan before the February resubmission. Deramiocel is an allogeneic cardiosphere-derived cell therapy designed to act via exosome secretion that targets macrophages to shift them toward a healing phenotype, but the committee concluded the available record did not meet the statutory threshold for effectiveness.