SAN DIEGO, June 26, 2026
Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has scheduled a Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) meeting for July 29, 2026 to review the company’s Biologics License Application (BLA) seeking approval of Deramiocel for the treatment of Duchenne muscular dystrophy (DMD). The regulatory review remains on schedule with a PDUFA target action date of August 22, 2026. The BLA is supported by data from the Phase 2 HOPE-2 trial, long-term findings from the HOPE-2 Open Label Extension (HOPE-2-OLE) study, and positive results from the pivotal Phase 3 HOPE-3 trial, which achieved statistical significance for its primary endpoint measuring Performance of the Upper Limb (PUL v2.0), the key secondary endpoint assessing left ventricular ejection fraction (LVEF), and all other Type I error-controlled secondary endpoints. If approved, Deramiocel could become a first-in-class cell therapy for patients living with DMD.
Clinical Data Supports Deramiocel BLA
Capricor’s regulatory submission is built on multiple clinical studies demonstrating clinically meaningful improvements in both skeletal and cardiac muscle function. The company reported that HOPE-3, its Phase 3 registrational study, successfully met its primary and secondary efficacy endpoints while maintaining a consistent safety profile observed across previous studies. Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that work by releasing exosomes, extracellular vesicles that modulate immune activity and reduce inflammation by promoting tissue repair mechanisms. Through immunomodulatory and anti-fibrotic effects, the therapy aims to preserve muscle function and slow disease progression in Duchenne muscular dystrophy, a rare genetic disorder caused by the absence of functional dystrophin, resulting in progressive muscle degeneration affecting skeletal, respiratory, and cardiac muscles.
FDA Review Marks Important Regulatory Milestone
The upcoming FDA Advisory Committee meeting provides an opportunity for independent experts to evaluate the complete clinical evidence supporting Deramiocel before the agency makes its final regulatory decision. The meeting will include discussions involving FDA reviewers, clinicians, researchers, and members of the DMD patient community, with public live streaming available. Capricor stated that the company remains focused on supporting the FDA’s ongoing review process while preparing for the advisory committee meeting. Deramiocel has already received several important regulatory designations, including Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), Regenerative Medicine Advanced Therapy (RMAT) designation in the United States, Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation, which could make the company eligible for a Priority Review Voucher if approval is granted.
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Expanding Cell and Exosome-Based Therapies for Rare Diseases
Beyond Deramiocel, Capricor continues to advance its broader pipeline of cell and exosome-based therapeutics for rare diseases. The company’s proprietary StealthX™ exosome platform is being developed to deliver oligonucleotides, proteins, and small-molecule therapeutics with targeted precision across multiple disease areas. While Deramiocel remains Capricor’s most advanced clinical program, the company believes its exosome technology could support future therapies for a wide range of serious conditions. With the FDA Advisory Committee review scheduled for July 29, 2026, followed by the August 22, 2026 PDUFA decision, the coming months represent a significant regulatory milestone that could potentially introduce a new treatment option for patients affected by Duchenne muscular dystrophy.
Source: Capricor Therapeutics press release
