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Getting the latest healthcare news for you
Getting the latest healthcare news for you
The FDA has extended its review of deramiocel — an investigational cell therapy for Duchenne muscular dystrophy (DMD) — by three months to November 22, 2026, after accepting new HOPE-3 trial data as a major amendment. This comes despite an FDA advisory committee voting 9-3 against recommending approval in July, citing disputes over the trial's statistical analysis plan and questions about cardiac endpoints.
The FDA has pushed back its decision deadline on deramiocel, Capricor Therapeutics' investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22 to November 22, 2026. The extension was triggered by the agency's acceptance of a major amendment to the biologics license application (BLA), which includes 24-month open-label extension data and additional robustness analyses from the phase 3 HOPE-3 trial. Capricor submitted the amendment after a July 2026 FDA advisory committee meeting and is now seeking a refined indication focused on preserving upper limb function.
The road to this point has been rocky. An FDA advisory committee voted 9-3 against recommending approval in July, with panelists questioning Capricor's revisions to the trial's statistical analysis plan (SAP) after randomization. The FDA and Capricor clashed over which SAP version was "prespecified" — the FDA used version 1.1 and found no significant treatment effect, while Capricor used version 3.0 and reported a statistically significant slowing of upper limb decline. The committee also raised concerns about whether left ventricular ejection fraction (LVEF) is a validated surrogate for clinical benefit.
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Why it matters: DMD remains incurable, and progressive cardiomyopathy is a leading cause of death in these patients. The extended review keeps hope alive for a therapy with no approved alternatives for this complication, but the path to approval remains uncertain given the advisory committee's skepticism and unresolved statistical disputes.
