FDA Advisory Panel Recommends Against Deramiocel for DMD Cardiomyopathy

Capricor Therapeutics announced that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee voted 3 in favor and 9 against, with no abstentions, that the available evidence did not support the effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). The advisory committee’s recommendation is non-binding, and the FDA is expected to issue its final regulatory decision by the August 22, 2026 Prescription Drug User Fee Act (PDUFA) target action date.

According to Capricor, the committee’s voting question addressed a narrower indication than the company had proposed and did not include an assessment of Deramiocel’s overall benefit-risk profile. In a separate discussion on upper limb function, committee members provided directionally supportive feedback on the clinical evidence from the Phase 3 HOPE-3 trial (NCT05126758), including results for the study’s primary endpoint, Performance of Upper Limb (PUL) 2.0.

Company remains confident in HOPE-3 data despite advisory committee vote

Capricor stated that it remains committed to advancing Deramiocel and continues to believe that the totality of clinical evidence supports the therapy’s potential benefit. The company highlighted the committee’s discussion regarding preservation of skeletal muscle function and reiterated its confidence in the strength of the Phase 3 HOPE-3 data package.

Chief Executive Officer Linda Marbán, Ph.D., said the advisory committee meeting provided an important opportunity to present the clinical evidence supporting Deramiocel. She noted that the discussion on skeletal muscle outcomes was encouraging and emphasized that patients, families, and clinicians who participated in the open public hearing highlighted the significant unmet medical need within the Duchenne community. The company stated that it remains focused on working with the FDA as the review process continues ahead of the August 22, 2026 PDUFA action date.

The Lancet publication provides independent peer-reviewed support for HOPE-3 findings

On the same day as the FDA Cellular, Tissue and Gene Therapies Advisory Committee meeting, Capricor also announced that The Lancet had published the results of the pivotal Phase 3 HOPE-3 trial evaluating Deramiocel in patients with Duchenne muscular dystrophy. According to the company, the peer-reviewed publication provides independent validation of the trial’s design, statistical methodology, and findings based on the prespecified Statistical Analysis Plan version 3.0.

The published study, titled “Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial,” reported that Deramiocel significantly slowed the decline in upper limb function compared with placebo, meeting the trial’s primary endpoint of Performance of Upper Limb (PUL) 2.0. The publication complements the company’s position that the totality of clinical evidence supports Deramiocel’s potential benefit as the FDA continues its review ahead of the August 22, 2026, PDUFA target action date.

DMD-associated cardiomyopathy remains a major unmet medical need

Duchenne muscular dystrophy is a rare, severe X-linked genetic disorder characterized by progressive degeneration of skeletal, respiratory, and cardiac muscles due to the absence of functional dystrophin. The disease primarily affects boys and impacts approximately 15,000 individuals in the United States.

As DMD progresses, deterioration of the heart muscle can lead to cardiomyopathy and heart failure, which represent the leading causes of death in affected patients. Despite advances in supportive care, there are currently no FDA-approved therapies specifically indicated for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy, highlighting a significant unmet medical need.

Deramiocel designed to preserve cardiac and skeletal muscle function

Deramiocel (CAP-1002) is an investigational allogeneic cardiosphere-derived cell (CDC) therapy being developed for Duchenne muscular dystrophy. CDCs are a rare population of cardiac cells that have demonstrated immunomodulatory and anti-fibrotic effects in preclinical and clinical studies and are intended to help preserve both cardiac and skeletal muscle function.

According to Capricor, the therapeutic activity of CDCs is mediated primarily through the secretion of extracellular vesicles known as exosomes, which interact with macrophages and promote a healing rather than pro-inflammatory phenotype. The company noted that CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 participants across multiple clinical studies.

Multiple regulatory designations support Deramiocel’s development

Deramiocel has received several regulatory designations recognizing its potential role in treating Duchenne muscular dystrophy. The therapy has been granted Orphan Drug Designation by 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.

Capricor noted that approval of Deramiocel could make the company eligible to receive a Priority Review Voucher under the FDA’s Rare Pediatric Disease program. The company stated that it remains committed to working with regulators to advance Deramiocel for patients living with Duchenne muscular dystrophy who currently have limited treatment options for DMD-associated cardiomyopathy.

Reference

Capricor Therapeutics Provides Update on FDA Advisory Committee Meeting for Deramiocel :: Capricor Therapeutics, Inc. (CAPR)