Capricor’s Deramiocel BLA Review Reinstated by FDA Following HOPE-3 Trial Data

Capricor Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has resumed review of the company’s Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The application has been accepted as a Class 2 resubmission following the company’s response to a previously issued Complete Response Letter (CRL) in July 2025, with a Prescription Drug User Fee Act (PDUFA) target action date set for August 22, 2026.

The regulatory update follows Capricor’s submission of additional data and supporting documentation from the Phase 3 HOPE-3 clinical trial. According to the company, the FDA has resumed review of the application and has not identified any potential review issues at this stage.

Results from the Phase 3 HOPE-3 trial (NCT05126758) showed that Deramiocel achieved statistically significant benefits across both skeletal and cardiac endpoints. The therapy slowed disease progression by 54 percent on the Performance of Upper Limb (PUL v2.0) total score, the study’s primary endpoint, compared with placebo, demonstrating preservation of upper limb function in both ambulatory and non-ambulatory patients. In addition, Deramiocel showed 91 percent preservation of left ventricular ejection fraction (LVEF) versus placebo, the study’s key secondary endpoint, highlighting a potential protective effect on cardiac function. Cardiomyopathy remains the leading cause of death in patients with DMD.

“We are encouraged by the FDA’s acknowledgment of our response to the Complete Response Letter and its continued review of our BLA for Deramiocel,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor Therapeutics. She added that the HOPE-3 results, along with broader clinical evidence, reinforce the potential of Deramiocel to become a first-in-class therapy for Duchenne muscular dystrophy, with the ability to address both skeletal and cardiac manifestations of the disease.

Capricor previously received a CRL from the FDA in July 2025. Following submission of the additional HOPE-3 data and supporting materials, the agency accepted the resubmission and assigned a new PDUFA action date of August 22, 2026.

Duchenne muscular dystrophy is a severe X-linked genetic disorder caused by the absence of functional dystrophin, a protein essential for maintaining muscle cell structure. The disease leads to progressive degeneration of skeletal, respiratory, and cardiac muscles and primarily affects boys. Approximately 15,000 individuals in the United States are estimated to be living with DMD, and treatment options remain limited.

Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a specialized population of cardiac cells shown in preclinical and clinical studies to exert immunomodulatory and anti-fibrotic effects that may help preserve cardiac and skeletal muscle function in muscular dystrophies such as DMD. These cells act largely through the secretion of extracellular vesicles called exosomes, which interact with macrophages and shift them toward a reparative, anti-inflammatory phenotype.

The CDC platform has been studied extensively, with more than 250 peer-reviewed scientific publications and administration in over 250 patients across multiple clinical trials.

Deramiocel has received several regulatory designations supporting its development, 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 from the FDA. If approved, the therapy may also qualify Capricor for a Priority Review Voucher.

 References

Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA, 10 March 2026, https://www.capricor.com/investors/news-events/press-releases/detail/338/capricor-therapeutics-announces-establishment-of-new-pdufa

Positive HOPE-3 Phase 3 Results Support Deramiocel as a Potential New Treatment for Duchenne Muscular Dystrophy, 05 December 2026, https://pharmacally.com/positive-hope-3-phase-3-results-support-deramiocel-as-a-potential-new-treatment-for-duchenne-muscular-dystrophy/

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy (HOPE-3), ClinicalTrials.gov ID NCT05126758, https://clinicaltrials.gov/study/NCT05126758