The U.S. Food and Drug Administration (FDA) will take three more months to decide whether to approve deramiocel (CAP-1002), an experimental therapy for Duchenne muscular dystrophy (DMD), setting a new target decision date of Nov. 22, 2026, developer Capricor Therapeutics announced Aug. 24.
The new timeline follows Capricor’s submission of new data meant to support a narrower proposed use for the therapy, focused on preserving upper limb function — the ability to use the arms and hands. It comes after a July advisory committee meeting where a panel of independent experts questioned deramiocel’s evidence for treating the heart complications of DMD but responded more favorably to its effects on arm and hand movement.
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While many associate DMD mainly with muscle weakness in the limbs, over time it also weakens the heart. This can cause cardiomyopathy, the leading cause of death in DMD. Some therapies are approved for DMD, but none for its heart complications.
Capricor said the amendment to its application includes two years of follow-up data from its Phase 3 HOPE-3 study, the trial in which upper limb function was the main measure of success, along with additional analyses. The FDA’s Center for Biologics Evaluation and Research accepted the submission for review, citing the significant unmet medical need in DMD, but classified it as a major amendment — a designation that added three months to the review timeline.
Linda Marbán, chief executive officer of Capricor, said HOPE-3 met its primary goal, with treated patients showing a statistically significant improvement in arm and hand function. She noted that in a progressive disease like DMD, “function, once lost, cannot be recovered,” and said protecting that function could make a real difference to patients’ independence and everyday lives.
Deramiocel is an experimental cell therapy made from cardiosphere-derived cells, which studies suggest can reduce inflammation and scarring while helping to preserve muscle function. It has not been approved for use in any country.
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