Capricor Therapeutics’ resubmitted biologics license application for Deramiocel took another hit after the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee recommended against approval. In a 9–3 vote, the panel said the Phase 3 HOPE-3 evidence for Deramiocel did not provide “substantial evidence of effectiveness” for cardiomyopathy in Duchenne muscular dystrophy. The advisory committee’s view was shaped by the record from HOPE-3 (NCT05126758) and earlier HOPE-2 data, alongside concerns raised in the FDA review process. FDA staff questioned endpoint performance, noted hypersensitivity signals of 42% versus 15% with placebo, and cited the absence of an updated statistical analysis plan before the February BLA resubmission. Capricor now faces an FDA target decision date of August 22 under PDUFA, with the panel outcome likely to factor into how the agency weighs its resubmission despite the non-binding nature of adcomm votes.