The US FDA has resumed review of a Biologics License Application (BLA) from Capricor Therapeutics for Deramiocel (CAP-1002), an allogeneic cardiosphere-derived cell therapy for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy, with a PDUFA target action date of August 22, 2026. The agency lifted a Complete Response Letter issued in July 2025 after Capricor submitted additional data from the HOPE-3 Phase 3 trial. If approved, Deramiocel would be the first therapy to address both skeletal and cardiac manifestations of DMD and the first cell therapy approved for the disease. Capricor has said it expects to be eligible for a Priority Review Voucher upon approval, given the product's Rare Pediatric Disease Designation.
The submission has been classified as a Class 2 resubmission. The BLA seeks full approval rather than accelerated approval, distinguishing it from the regulatory pathway used for the exon-skipping antisense oligonucleotides and the AAV-based gene therapy delandistrogene moxeparvovec (Elevidys), all of which received accelerated approval based on surrogate endpoints. The US FDA has not identified potential review issues in its initial response to the resubmission, the company said. Deramiocel holds Orphan Drug Designation from both the US FDA and the European Medicines Agency, as well as Regenerative Medicine Advanced Therapy designation in the United States.
The BLA is supported by data from the HOPE-3 trial, a Phase 3 study in which Deramiocel met its primary endpoint of change from baseline in mid-level Performance of the Upper Limb (PUL 2.0) score at 12 months compared to placebo. All Type I error-controlled secondary endpoints were also met, the company said, though detailed effect sizes, confidence intervals, and full safety data have not yet been disclosed in a peer-reviewed publication. Cardiosphere-derived cells, the active component of Deramiocel, have been administered to more than 250 subjects across multiple clinical trials, according to Capricor. The cells act by secreting exosomes that reprogram macrophages from a pro-inflammatory to a reparative phenotype, exerting immunomodulatory and anti-fibrotic effects on both cardiac and skeletal muscle tissue.
DMD is a severe X-linked genetic disorder affecting approximately 15,000 individuals in the United States, caused by the absence of functional dystrophin. Cardiomyopathy is the leading cause of death, yet no approved therapy has specifically targeted the cardiac component of the disease. Current cardiac management relies on ACE inhibitors, beta-blockers, and mineralocorticoid receptor antagonists borrowed from general heart failure treatment. The broader DMD treatment landscape includes corticosteroids as the standard of care, exon-skipping therapies from Sarepta Therapeutics and NS Pharma that collectively cover roughly 30% of patients with amenable mutations, and Elevidys, a one-time gene therapy with broad mutation coverage but unresolved questions about long-term durability. Deramiocel adds to this landscape as a mutation-agnostic approach with a distinct mechanism that does not attempt to restore dystrophin but instead targets the inflammatory and fibrotic processes driving disease progression. Capricor has entered into an exclusive commercialization agreement with Nippon Shinyaku for the United States and Japan, subject to regulatory approval.
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