FDA Advisory Committee Schedules Meeting to Review Deramiocel's BLA in Duchenne Muscular Dystrophy

Months after the FDA rescinded its complete response letter (CRL), the agency's Cellular, Tissue, and Gene Therapies Advisory Committee is scheduled to review Capricor Therapeutics' biologics license application (BLA) for deramiocel, an investigational allogeneic cardiac-derived cell therapy for Duchenne muscular dystrophy (DMD), on July 29, 2026.¹

“We are encouraged by the opportunity to bring Deramiocel before the Advisory Committee and engage directly with the FDA, the DMD patient community, and the physicians who care for them. We have confidence in the totality of evidence supporting Deramiocel, which has demonstrated clinically meaningful, statistically significant skeletal and cardiac benefits with a consistent safety profile, across multiple studies supporting its potential as a first-in-class therapy for Duchenne muscular dystrophy,” Linda Marbán, PhD, chief executive officer at Capricor, said in a statement.¹

In the company’s issued CRL in July 2025, the agency stated that it was unable to review the BLA because it did not meet the requirements for substantial evidence of efficacy.² In addition, the FDA noted outstanding items in the Chemistry, Manufacturing, and Controls section of the application, most of which the company stated it believed had been addressed in prior communications with the FDA. For context, the original BLA submission for deramiocel was based on data from the HOPE-2 trial (NCT03406780), its open-label extension (NCT04428476), and natural history comparisons from FDA-funded datasets.

HOPE-2 enrolled 26 patients with DMD cardiomyopathy, 8 who were randomized to deramiocel, 12 to placebo, and 6 who were excluded due to screening failure. All told, deramiocel-treated patients showed a statistically significant 36.2% improvement over placebo in 12-month mid-level elbow PUL1.2 scores (mean difference, 2.6 points; 95% CI, 12.7-59.7; P = .014). Treatment with the cell therapy led to significant reductions in creatine kinase (CK-MB) as a proportion of total CK over 12 months, indicating less cardiac muscle damage, with a 29.1% difference vs placebo (95% CI, 4.0-54.2; P = .025).³

READ MORE: BridgeBio Submits NDA for BBP-418 in LGMD2I/R9, Potentially the First Approved Therapy for Any Form of Limb-Girdle Muscular Dystrophy

The FDA made the decision to continue its review of the BLA in response to newly submitted data and supporting documentation from the ongoing phase 3 HOPE-3 clinical trial (NCT05126758).⁴ HOPE-3 is a randomized, double-blind, placebo-controlled, phase 3 trial testing deramiocel in boys and young men living with DMD across 20 sites in the United States. In the trial, participants received intravenous deramiocel at 150 million cells per infusion or placebo every 3 months for 12 months. Overall, the mean age of participants was approximately 15 years, and all were on a stable corticosteroid regimen during the trial.

Topline data from HOPE-3 demonstrated that treatment with deramiocel significantly delayed disease progression compared with placebo on Performance of Upper Limb total score (PUL v2.0), showing a 54% slowing of progression and meeting the primary end point in the intent-to-treat population (ITT) at 12 months (n = 105; P = .029).⁵ For the key secondary end point, left ventricular ejection fraction, deramiocel treatment was associated with a 91% slowing of decline based on centrally reviewed cardiac MRI evaluations in the ITT population across 12 months (n = 83; P = .041). Notably, the company observed that the safety and tolerability of deramiocel were consistent with prior studies in participants.

Deramiocel consists of allogeneic cardiosphere-derived cells. The proposed mechanism is not dystrophin replacement; rather, the cells are described as exerting immunomodulatory and antifibrotic effects through secretion of extracellular vesicles, including exosomes, that influence macrophage phenotype and inflammatory signaling. The cell therapy has previously received FDA orphan drug, regenerative medicine advanced therapy, and rare pediatric disease designations for DMD, as well as orphan drug and advanced therapy medicinal product designations in Europe.

DMD is an X-linked dystrophinopathy characterized by progressive degeneration of skeletal, respiratory, and cardiac muscle.⁶ Contemporary care is multidisciplinary and includes corticosteroids, pulmonary surveillance and ventilatory support, cardioprotective therapy, rehabilitation, orthopedic management, and mutation-directed therapies for eligible subsets of patients. Despite therapeutic advances, progressive cardiomyopathy and respiratory complications remain major contributors to morbidity and mortality, and functional decline in nonambulatory patients remains an area of substantial unmet need.

REFERENCES
1. Capricor Therapeutics announces FDA advisory committee meeting to review BLA for deramiocel for the treatment of Duchenne muscular dystrophy. Capricor Therapeutics. June 26, 2026. Accessed June 29, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/348/capricor-therapeutics-announces-fda-advisory-committee
2. Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy. News release. Capricor Therapeutics. July 11, 2025. Accessed June 29, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/319/capricor-therapeutics-provides-regulatory-update-on
3. McDonald CM, Marbán E, Hendrix S, et al. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet. 2022;399(10329):1049-1058. doi:10.1016/S0140-6736(22)00012-5
4. Capricor Therapeutics announces establishment of new PDUFA date for Deramiocel BLA. News release. Capricor Therapeutics. March 10, 2026. Accessed June 29, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/338/capricor-therapeutics-announces-establishment-of-new-pdufa
5. Capricor Therapeutics Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy. News release. Capricor Therapeutics. December 3, 2025. Accessed June 29, 2026. https://www.capricor.com/investors/news-events/press-releases/detail/331/capricor-therapeutics-announces-positive-topline-results
6. Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267. doi:10.1016/S1474-4422(18)30024-3