Capricor Therapeutics’ resubmitted biologics license application for Deramiocel took another hit after the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted against approval. In a 9–3 recommendation, the panel concluded that evidence from the Phase III HOPE-3 trial (NCT05126758) did not provide substantial evidence of effectiveness for Deramiocel in Duchenne muscular dystrophy (DMD) cardiomyopathy. The advisory committee decision is expected to weigh heavily on the FDA’s August 22 target action date under Prescription Drug User Fee Act (PDUFA), with the agency typically heeding adcom input. Deramiocel is an allogeneic cardiosphere-derived cell therapy designed to act via secreted exosomes that modulate macrophage behavior toward a healing phenotype. FDA reviewers raised multiple concerns ahead of the vote, including whether HOPE-3 endpoints were met and a hypersensitivity signal reported in 42% of patients versus 15% with placebo. The agency also criticized the lack of an updated statistical analysis plan (SAP) submitted prior to the February BLA resubmission. For DMD-focused developers, the setback underscores how tightly the FDA is scrutinizing clinical evidence quality—especially for surrogate immunologic mechanisms—when deciding whether confirmatory datasets support approval.