FDA Extends Deramiocel Review to November 2026 as Capricor Refines DMD Indication

The U.S. Food and Drug Administration (FDA) has extended the PDUFA target action date for Capricor Therapeutics’ investigational cell therapy deramiocel (CAP-1002) from August 22 to November 22, 2026, giving the agency additional time to review new Phase 3 HOPE-3 data and analyses supporting a refined proposed indication for Duchenne muscular dystrophy (DMD).

FDA Requests More Time to Review HOPE-3 Data

The extension follows Capricor’s submission of an amendment to its Biologics License Application (BLA) containing 24-month open-label extension data from HOPE-3, along with additional robustness analyses. The company is proposing an indication focused on upper limb function, which served as the pivotal trial’s primary endpoint.

The FDA’s Center for Biologics Evaluation and Research (CBER) accepted the amendment for review and classified it as a major amendment. The agency extended the review period by three months to allow sufficient time to assess the additional information, citing the significant unmet medical need in DMD.

The regulatory decision does not represent an approval of deramiocel. The therapy remains investigational while the FDA completes its review.

A Regulatory Reprieve for Deramiocel

The FDA’s decision provides some relief for Capricor after a difficult regulatory path for deramiocel. The therapy previously received a Complete Response Letter and, more recently, faced a challenging July advisory committee meeting, where members voted 9-3 against the available evidence supporting its proposed use. Although the vote was nonbinding, it added uncertainty around the program. Against this backdrop, the FDA’s acceptance of new 24-month HOPE-3 data and its decision to extend the review by three months give Capricor an opportunity to address those concerns under a refined indication focused on upper limb function. The extension does not assure approval, but it keeps the application under active review and gives the company additional time to make its clinical case.

HOPE-3 Supports Upper Limb Function

HOPE-3 is a pivotal Phase 3 study evaluating deramiocel in patients with DMD. According to Capricor, the trial met its primary endpoint and demonstrated a statistically significant benefit in upper limb function.

The newly submitted 24-month open-label data provide an additional year of follow-up beyond the previously available dataset. Capricor said the longer-term results and robustness analyses strengthen the evidence supporting preservation of upper limb function.

The focus on upper limb function has important clinical relevance in DMD, where progressive muscle degeneration can gradually impair mobility, respiratory function and the ability to perform daily activities independently. Once upper limb function is lost, recovery is generally not possible, making preservation an important treatment goal.

The announcement did not provide new numerical efficacy or safety data from the 24-month analysis. Detailed safety findings were also not disclosed in the update.

Cell Therapy Targets Muscle Degeneration

Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs), a population of cardiac-derived cells that has demonstrated immunomodulatory and anti-fibrotic activity in preclinical and clinical studies.

The cells release extracellular vesicles, including exosomes, that interact with macrophages and can shift their activity away from a pro-inflammatory state toward a tissue-repair phenotype. Capricor is developing the therapy to preserve skeletal and cardiac muscle function in DMD.

DMD is an X-linked genetic disorder caused by the absence of functional dystrophin, leading to progressive degeneration of skeletal, respiratory and cardiac muscle. Cardiomyopathy and heart failure remain major causes of mortality, while treatment options remain limited and no cure is currently available.

Regulatory Decision Expected in November

Capricor Chief Executive Officer Linda Marbán said the additional HOPE-3 follow-up provides an extensive dataset on upper limb function and argued that preservation of this function could have meaningful implications for independence and daily living.

Deramiocel has received Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease designations in the United States, as well as Orphan Drug and Advanced Therapy Medicinal Product designations in Europe.

The FDA now expects to complete its review by November 22, 2026. The decision will determine whether the available clinical and regulatory evidence supports licensure of deramiocel for DMD under the refined proposed indication.

Reference

Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA :: Capricor Therapeutics, Inc. (CAPR)