The U.S. Food and Drug Administration (FDA) has extended its review of Capricor Therapeutics’ investigational cell therapy deramiocel for Duchenne muscular dystrophy (DMD) by three months. The agency’s new Prescription Drug User Fee Act (PDUFA) target action date is November 22, replacing the previous deadline of August 22.
The extension follows Capricor’s submission of an amendment to its biologics license application (BLA). The amendment includes 24-month open-label extension data from the Phase 3 HOPE-3 study, along with additional analyses intended to support a revised proposed indication centered on upper limb function. The FDA classified the submission as a “major amendment,” resulting in the extended review period.
The company stated that the amendment asks the agency to evaluate both previously submitted and newly generated data in support of the refined indication. Upper limb function was the primary endpoint of the HOPE-3 trial.
Amendment Submitted After Advisory Committee Review
Last month, an FDA advisory committee voted 9-3 against recommending approval of deramiocel. During the meeting, regulators and advisers spent considerable time discussing cardiac-related data from the study.
Deramiocel has also been proposed as a treatment for DMD cardiomyopathy. Prior to submission of the BLA, Capricor requested during a pre-BLA meeting that the primary efficacy endpoint of HOPE-3 be changed from upper limb function to left ventricular ejection fraction (LVEF), a measure of heart function. The company subsequently submitted a protocol amendment seeking that change, but no modification was ultimately made. Upper limb function remained the primary endpoint, while LVEF remained a pre-specified secondary endpoint.
At the advisory committee meeting, members described the cardiac-related data associated with the secondary endpoint as “very fragile.” The committee’s vote against approval was based on the therapy’s efficacy data in DMD cardiomyopathy.
Submission Focuses on Upper Limb Function Data
Capricor Chief Executive Officer Linda Marbán has said that the emphasis on cardiac outcomes was misplaced because LVEF was a secondary endpoint. She also stated that the analysis discussed during the advisory committee meeting was conducted across the full DMD population rather than in patients with established cardiomyopathy, the population addressed by the proposed indication.
The company’s amendment requests FDA review of existing and new data supporting a refined proposed indication focused on upper limb function. Discussing the additional data submitted to the FDA, Marbán said: “HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function, and we believe the additional open-label data and further analyses included in the amendment strengthen the evidence supporting a refined proposed indication.”
Marbán also said the company appreciates the FDA’s continued engagement and looks forward to working with the agency as it completes its review.
Previous Regulatory Setbacks and Shareholder Concerns
Deramiocel previously did not receive FDA approval for DMD cardiomyopathy after the agency determined that the treatment did not meet efficacy requirements and requested additional data. Earlier this year, Capricor resubmitted its application with new information and had expected a decision by August 22.
Following the announcement of the review extension, Capricor shares rose. One report said the stock increased as much as 16% and was later valued at $7.05 per share, while another reported a gain of about 15% to $7.25.
The regulatory review also comes amid shareholder concerns. Investment firm Kaos Capital recently objected to Capricor’s plan to halt all other pipeline work until it receives further regulatory clarity on deramiocel. In a letter to shareholders, the firm stated that it believes deramiocel may still have meaningful value for patients and also cited the potential of Capricor’s cell-therapy and exosome capabilities, while arguing that the company should not concentrate all of its resources on a single regulatory outcome.
The FDA is expected to complete its review of deramiocel by November 22.
Capricor Therapeutics is facing another delay in the U.S. regulatory review of deramiocel, its investigational treatment for Duchenne muscular dystrophy (DMD). The FDA has extended the review period, with a new action date expected in November 2026. The decision gives regulators additional time to evaluate the company’s application and supporting information.
Capricor and Deramiocel Review
The Capricor application seeks approval for deramiocel as a potential treatment for boys and young men living with Duchenne muscular dystrophy. The therapy is designed to address the progressive muscle damage associated with the disease.
The extended review means the FDA will have additional time to assess the application before reaching a regulatory decision.
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