FDA Grants Priority Review to Deramiocel for DMD Cardiomyopathy

The US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) filing for deramiocel, an investigational cell therapy targeted for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy.1

Announced by Capricor Therapeutics, Inc. on March 4, 2025, deramiocel has been granted Priority Review, with an assigned Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. Capricor announced that the regulatory agency has yet to determine whether an Advisory Committee meeting is needed for the BLA.

“We are thrilled to announce the acceptance of our BLA bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies,” said Linda Marban, PhD, chief executive officer of Capricor.1 “If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landsape.”

A genetic disorder defined by the progressive weakening and chronic inflammation of the skeletal, heart, and respiratory muscles, individuals with DMD experience mortality at a median of 30 years of age. It is estimated to occur in one in every 3500 male births with a population of 15,000–20,000 estimated in the US.2

Impaired production of function dystrophin in muscle cells can severely damage and cause muscle cell death—for patients with DMD, heart muscle cells progressively die and get replaced by scar tissue. Cardiomyopathy can progress to heart failure, the leading cause of mortality for individuals with DMD.

With limited treatment options, deramiocel could become a first-in-class therapeutic for DMD-cardiomyopathy, pending FDA approval.1

“Deramiocel has shown in multiple clinical trials attenuation of DMD-cardiomyopathy, which is currently one of the leading causes of death in those with DMD,” said Craig McDonald, MD, a professor and chair of the department of physical medicine and rehabilitation at the University of California, Davis.1 “Based on the totality of the safety and efficacy data deramiocel has shown, this potential approval offers patients a first-in-class therapeutic for DMD-cardiomyopathy.”

Capricor’s BLA submission was supported by existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 open-label extension trials, compared with natural history data from an FDA-funded dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression.

The HOPE-2 trial assigned 8 male patients with DMD to deramiocel and 12 to the placebo group, with 6 patients not randomized due to screening failure. Cardiac MRI assessments in HOPE-2 demonstrated heart function and structure improvement with deramiocel treatment.3

Compared with placebo, deramiocel improved ranked change in left ventricular ejection fraction (LVEF), with a statistically significant difference in percentile ranks of 45.7 (95% CI, 19.1–72.2). Other exploratory cardiac MRI structure endpoints also revealed improvement with deramiocel, compared with placebo, including reductions in indexed left ventricular end-systolic (LVESV) and end-diastolic volumes (LVEDV).

In the HOPE-2 open-label extension study, deramiocel continued to improve cardiac measures. Higher improvements in cardiac function were identified in patients with higher EFs (>45%) at the end of the HOPE-2 trial, with +3.0% improvement in LVEF and –5.1 and –10.0 mL/m2 decreases in indexed LVESC and LVEDV, respectively, at the 3-year timepoint.4

In January 2022, Capricor and Nippon Shinyaku agreed on exclusive distribution for deramiocel in the US market. A subsidiary of Nippon Shinyaku, NS Pharma Inc., was announced as exclusively responsible for the commercialization and distribution of deramiocel in the US.

“Deramiocel has the potential to address a clear, unmet medical need for patients diagnosed with DMD," said Yukiteru Sugiyama, PhD, president of NS Pharma.5 "We are excited for the possibility to bring additional treatment options and renewed hope to families of the rare disease community."

References

1. Capricor Therapeutics announces FDA acceptance and Priority Review of its biologics license application for Deramiocel to treat Duchenne muscular dystrophy. Capricor Therapeutics, Inc. March 4, 2025. Accessed March 10, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority.

2. Venugopal V, Pavlakis S. Duchenne Muscular Dystrophy. [Updated 2023 Jul 10]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK482346/

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 long-term benefit of Deramiocel (cap-1002) in both skeletal muscle and cardiac function in the HOPE-2 Ole Study in Duchenne muscular dystrophy. Capricor Therapeutics, Inc. June 28, 2024. Accessed March 10, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/284/capricor-therapeutics-announces-long-term-benefit-of.

5. Sultan K. FDA accepts Biologics License Application for duchenne muscular dystrophy cardiomyopathy treatment. NSPharma. March 6, 2025. Accessed March 10, 2025. https://www.nspharma.com/fda-accepts-biologics-license-application-for-duchenne-muscular-dystrophy-cardiomyopathy-treatment/.