The US FDA has extended its review of Capricor Therapeutics' (Nasdaq: CAPR) deramiocel BLA by three months after accepting a major amendment that refines the proposed Duchenne muscular dystrophy indication around preservation of upper limb function, weeks after an advisory committee voted against the cell therapy's effectiveness for DMD cardiomyopathy.
The agency moved the PDUFA date from August 22 to November 22, 2026, after Capricor submitted 24-month open-label extension data from the Phase III HOPE-3 study and additional analyses supporting the refined indication. The FDA classified the submission as a major amendment, allowing the review to continue under the existing BLA rather than requiring a new resubmission.
In July 2026, the FDA's Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3 against the effectiveness of deramiocel for DMD cardiomyopathy — the indication originally sought — though the committee's feedback on upper limb function was described by Capricor as directionally supportive of the HOPE-3 clinical evidence. The committee's vote is non-binding, and the FDA retains full authority to approve or reject the application independently. Capricor's decision to submit an amendment narrowing the proposed indication to upper limb function came in response to the committee's divided assessment.
The pivotal HOPE-3 trial, a randomized, double-blind, placebo-controlled Phase III study enrolling 106 patients with DMD, met its primary endpoint of change from baseline in Performance of the Upper Limb version 2.0 (PUL 2.0) score, with deramiocel slowing upper limb function decline by 54% versus placebo (p=0.029), according to data published in The Lancet on July 29, 2026. All Type I error-controlled secondary endpoints were also met, Capricor said. The 24-month open-label extension data now included in the BLA amendment add a longer follow-up period to the original 12-month controlled dataset, the company said, and are intended to strengthen the evidence base for a label focused specifically on upper limb preservation.
The FDA issued a Complete Response Letter in July 2025 based on the original submission. Following submission of the HOPE-3 clinical study report, the FDA resumed review in March 2026 as a Class 2 resubmission with the August 22 PDUFA date. Pre-meeting FDA briefing documents ahead of the July advisory committee raised questions about the strength of the cardiac evidence and the statistical methodology used in HOPE-3, including concerns about post-hoc modifications to the statistical analysis plan. The advisory committee's negative vote on cardiomyopathy — while directionally supportive on skeletal muscle outcomes — prompted Capricor to reframe the proposed indication around the endpoint the trial was designed and powered to detect.
Deramiocel consists of allogeneic cardiosphere-derived cells that secrete exosomes targeting macrophages, shifting them from a pro-inflammatory to a pro-healing phenotype to exert immunomodulatory and anti-fibrotic effects on skeletal and cardiac muscle. Unlike approved exon-skipping antisense oligonucleotides — which target specific mutations and collectively cover roughly 30% of DMD patients — deramiocel is mutation-agnostic and acts on downstream inflammatory and fibrotic pathways rather than attempting to restore dystrophin. No approved therapy is currently indicated specifically for upper limb function preservation in non-ambulatory DMD patients, the population most represented in HOPE-3. Sarepta Therapeutics' delandistrogene moxeparvovec (Elevidys), the only approved DMD gene therapy, delivers a truncated micro-dystrophin and has faced safety headwinds following pediatric deaths from acute liver failure in 2025.
Capricor holds Orphan Drug Designation from both the US FDA and the European Medicines Agency (EMA), as well as Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease Designations in the US — the latter of which may qualify the company for a transferable Priority Review Voucher upon approval. The company said it has administered approximately 1,300 intravenous infusions of deramiocel to over 200 patients across three clinical trials, with some patients receiving continuous infusions for more than five years. The FDA's final decision on the BLA is now expected by November 22, 2026.
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