Capricor Therapeutics said the FDA extended Deramiocel's PDUFA target action date to November 22, 2026, from August 22. The extension follows submission of 24-month open-label extension data from the Phase 3 HOPE-3 trial supporting a refined indication focused on upper limb function in Duchenne muscular dystrophy.
Capricor Therapeutics said the FDA extended Deramiocel's PDUFA date by three months to November 22, 2026, after submitting additional HOPE-3 data.
"With an additional year of follow-up from HOPE-3, we now have one of the most extensive clinical datasets evaluating upper limb function in Duchenne," Linda Marbán, chief executive officer at Capricor, said.
The amendment includes 24-month open-label extension data from the Phase 3 HOPE-3 trial and additional robustness analyses supporting a refined proposed indication focused on upper limb function, the trial's primary endpoint. HOPE-3 enrolled 106 boys and young men aged 10 and older with DMD, showing a 4.55 percent advantage over placebo in total Performance of Upper Limb 2.0 score (P=.029). The FDA's Center for Biologics Evaluation and Research classified the submission as a major amendment.
The extension follows a July advisory committee vote of 9-3 against the cardiomyopathy indication Capricor originally pursued. The refined indication narrows the therapy's scope to upper limb preservation, a shift that could affect the label's patient reach. The new PDUFA date of November 22 gives the agency three additional months to complete its review.
HOPE-3 also met key secondary cardiac endpoints. Treatment produced a statistically significant benefit in left ventricular ejection fraction, an absolute treatment difference of approximately 2.4 percent versus placebo (P=.041). Among patients with evaluable cardiac MRI scans, Deramiocel reduced progression of myocardial fibrosis by a mean of 3.00 affected cardiac segments (95% CI, -5.50 to -0.51; P=.022).
The FDA and Capricor disagree on how to interpret the upper limb data. Agency reviewers, using an earlier prespecified statistical plan, calculated a mean difference of 0.66 points with a p-value of 0.24, while Capricor's analysis under a later plan produced the 4.55 percent difference at P=.029. That statistical dispute was central to the July meeting of the Cellular, Tissue, and Gene Therapies Advisory Committee.
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells that secrete exosomes targeting macrophages to adopt a healing phenotype. The therapy holds Orphan Drug, RMAT and Rare Pediatric Disease designations in the U.S. DMD affects approximately 15,000 people in the United States, primarily boys, with cardiomyopathy a leading cause of death. Across studies, Deramiocel has maintained a favorable safety profile with no imbalance in serious adverse events between treatment and placebo groups in HOPE-3.
Capricor reported a second-quarter net loss of $40.7 million with $237.9 million in cash and marketable securities as of June 30. The company has slowed commercial readiness spending while awaiting the FDA decision. An FDA bioresearch monitoring inspection in July produced one Form 483 observation covering standard operating procedures and vendor oversight.
The three-month extension introduces regulatory uncertainty for Capricor shareholders, who now face a November 22 decision date instead of August 22. A positive outcome could make Deramiocel the first cell-based therapy approved for DMD, while the narrowed indication limits the label's scope to upper limb function.
This article is for informational purposes only and does not constitute investment advice.
