Capricor Therapeutics (NASDAQ: CAPR) Faces Its Prescription Drug User Fee Act (PDUFA) Target Action Date Saturday August 22, 2026 for Deramiocel (a Cardiosphere-Derived Cell Therapy) in the Treatment of Duchenne Muscular Dystrophy (DMD) Cardiomyopathy Following the July 29 FDA Cellular, Tissue, and Gene Therapies Advisory Committee 9-3 Vote Against Approval, With Panel Members Citing Concerns About the Stability of the Statistical Results and How Missing Data Were Handled in the Phase 3 HOPE-3 Trial Left Ventricular Ejection Fraction (LVEF) Endpoint Analysis; A Complete Response Letter (CRL) Is the Expected Outcome, and Would Mark the Second CRL for Deramiocel Following the July 2025 Initial Rejection That Also Cited Inadequate Substantial Evidence of Effectiveness
Capricor Therapeutics (NASDAQ: CAPR) faces its Prescription Drug User Fee Act (PDUFA) target action date Saturday August 22, 2026 for deramiocel (a cardiosphere-derived cell therapy, not a peptide) in the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. Background: the FDA issued a Complete Response Letter (CRL) in July 2025 citing that the Phase 2 data supporting the cell therapy fell short of the statutory requirement for substantial evidence of effectiveness. Capricor reported positive Phase 3 HOPE-3 trial results in December 2025 meeting both primary and secondary endpoints, and the FDA accepted the resubmission in March 2026 with a Class 2 resubmission classification and August 22 target action date. The July 29, 2026 FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that available evidence does not provide substantial evidence of effectiveness to recommend approval. Panel members cited concerns about the stability of the statistical results, saying that left ventricular ejection fraction (LVEF) endpoint outcomes appeared highly sensitive to how missing data were handled and which analytic assumptions were applied. A second Complete Response Letter is the expected outcome. Duchenne muscular dystrophy affects approximately 20,000 US patients (predominantly boys with X-linked inheritance) and progressive cardiomyopathy is the leading cause of death in the population. The DMD cardiomyopathy indication remains without an FDA-approved therapy.