FDA Schedules Advisory Committee Review of Deramiocel BLA for DMD

Capricor Therapeutics’ investigational cell therapy deramiocel heads to FDA advisory committee review on July 29, 2026, with a PDUFA decision expected August 22, 2026, for Duchenne muscular dystrophy.

Capricor Therapeutics has reached another major regulatory milestone as the U.S. Food and Drug Administration (FDA) scheduled a Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) meeting on July 29, 2026, to review the Biologics License Application (BLA) for deramiocel, an investigational allogeneic cell therapy for Duchenne muscular dystrophy (DMD). The BLA remains on track for its Prescription Drug User Fee Act (PDUFA) target action date of August 22, 2026.

The advisory committee will evaluate the totality of evidence supporting deramiocel, including results from the Phase 2 HOPE-2 trial (NCT03406780), its long-term open-label extension HOPE-2-OLE (NCT04428476), and the confirmatory Phase 3 HOPE-3 trial (NCT05126758). According to the company, HOPE-3 met its primary endpoint of Performance of the Upper Limb version 2.0 (PUL v2.0) while also achieving statistical significance for the key secondary endpoint of left ventricular ejection fraction (LVEF) and all other Type I error-controlled secondary endpoints.

Deramiocel Targets Progressive Muscle and Cardiac Damage

Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that exert immunomodulatory and anti-fibrotic effects through the release of extracellular vesicles, or exosomes. These exosomes reprogram inflammatory macrophages toward a tissue-repair phenotype, preserve both skeletal and cardiac muscle function in patients with DMD.

The therapy has been evaluated in more than 250 peer-reviewed scientific publications and administered to over 250 participants across multiple clinical studies.

Duchenne muscular dystrophy is a rare, X-linked genetic disorder caused by the absence of functional dystrophin. The disease leads to progressive skeletal muscle weakness, respiratory decline, and cardiomyopathy, with heart failure remaining the leading cause of death. Approximately 15,000 people in the United States are living with DMD, and effective treatment options remain limited.

Clinical Evidence Supports FDA Review

The BLA package combines evidence from randomized and long-term extension studies with confirmatory Phase 3 findings. Across these studies, deramiocel demonstrated clinically meaningful improvements in upper limb function while preserving cardiac performance, two critical measures of disease progression in non-ambulatory patients with DMD.

The company also reported a consistent safety profile across clinical development, supporting the therapy’s benefit-risk assessment as FDA reviewers prepare for the advisory committee meeting.

Company Highlights Confidence Ahead of Advisory Committee

Chief Executive Officer Linda Marbán, Ph.D., said the advisory committee meeting provides an opportunity to discuss the complete clinical evidence with FDA experts, physicians, and the Duchenne community. She noted that the clinical program demonstrated statistically significant improvements in both skeletal muscle and cardiac outcomes across multiple studies while maintaining a consistent safety profile, reinforcing deramiocel’s potential to become the first approved therapy in its class for DMD.

Regulatory Path Forward

The July 29 advisory committee meeting represents one of the final milestones before the FDA completes its review. While the committee’s recommendation is not binding, its assessment often plays an important role in regulatory decision-making.

Deramiocel has received Orphan Drug Designation from both the FDA and the European Medicines Agency, Regenerative Medicine Advanced Therapy (RMAT) designation in the United States, Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA. If approved, Capricor could also become eligible for a Priority Review Voucher, adding further strategic value to the program.

With the FDA decision expected on August 22, 2026, deramiocel could become a new therapeutic option for patients with Duchenne muscular dystrophy, addressing both skeletal muscle decline and progressive cardiac dysfunction through a novel cell-based mechanism. If approved, deramiocel would represent the first cell-based therapy to demonstrate durable preservation of both skeletal and cardiac muscle function in Duchenne muscular dystrophy.

Reference

Capricor Therapeutics Announces FDA Advisory Committee Meeting to Review BLA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy:: Capricor Therapeutics, Inc. (CAPR)