Capricor Therapeutics announced that an independent panel advising the U.S. Food and Drug Administration (FDA) voted that current evidence does not support the effectiveness of deramiocel for treating cardiomyopathy, a disease of the heart muscle, in people with Duchenne muscular dystrophy (DMD).
The vote, from the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee, is non-binding, meaning the agency does not have to follow it when making its final decision. The company said the FDA is expected to decide whether to approve the therapy by Aug. 22, 2026.
The panel concluded that neither the Phase 2 HOPE-2 study nor the Phase 3 HOPE-3 study provided sufficient evidence of deramiocel’s efficacy. However, in a separate discussion of arm and hand movement, the panel was more supportive. The company said members viewed the HOPE-3 data on upper limb function, measured by a scale known as PUL 2.0, more favorably.
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In DMD muscles throughout the body, including the heart muscle, progressively weaken because the body cannot make a working form of a key muscle protein called dystrophin. Several exon-skipping therapies, which help some patients produce a partial form of dystrophin, are approved to treat DMD, but no treatment is approved for the cardiomyopathy that the disease causes.
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Deramiocel is an experimental cell therapy made from cardiosphere-derived cells — a type of cell that studies suggest can reduce inflammation and scarring while helping to preserve heart and skeletal muscle function.
Capricor first submitted deramiocel for approval in December 2024, based on the Phase 2 HOPE-2 trial and interim results from its open-label extension study. In July 2025, the FDA issued a complete response letter, a notice that it will not approve a therapy in its current form and that spells out the issues a company must resolve.
FDA said the data had not met the legal standard for substantial evidence of effectiveness, meaning the studies had not sufficiently proven that the therapy works. Capricor later added data from its larger Phase 3 HOPE-3 trial, which led the FDA to resume its review.
