DMD Cell Therapy Delays Disease Progression in Phase III Trial
The therapy slowed arm-function loss about 54% and showed preserved heart function in a subgroup, while allergic reactions were more common, researchers said.
- On Wednesday, July 29, 2026, an FDA advisory panel voted 9-3 against Capricor Therapeutics' deramiocel, determining the data lacked "substantial evidence" of effectiveness for treating Duchenne muscular dystrophy cardiomyopathy.
- FDA scientists expressed skepticism about Capricor's data, arguing analysis plan changes created a "fragile" result that missed initial Phase 3 goals rather than demonstrating genuine clinical benefit.
- Defending the trial data on Monday, Capricor CEO Linda Marbán stated, "We are completely shocked at how they decided to review and analyze this data," while a Lancet study confirmed muscle-weakening slowdown.
- The FDA is expected to issue a final decision on deramiocel by August 22, 2026; the agency typically follows advisory panel recommendations, making this vote a significant setback for approval.
- This result marks the latest twist in an unusual regulatory saga, serving as a critical barometer for how flexible the FDA might be in assessing future rare disease drug submissions.
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FDA panel votes against Capricor’s Duchenne heart disease therapy
July 29 (Reuters) - The U.S. FDA's panel of independent advisers on Wednesday voted against the benefits of Capricor Therapeutics' cell therapy, deramiocel, for treating cardiomyopathy in Duchenne muscular dystrophy, a rare muscle-wasting disease. Th...
DMD Cell Therapy Delays Disease Progression in Phase III Trial
Cell therapies are growing in variety and effectiveness across many diseases. In the fight against Duchenne muscular dystrophy (DMD), deramiocel has emerged as an effective treatment for DMD in patients in early clinical trials. New data from the HOPE-3 Phase III clinical trial, published in The Lancet, expand the efficacy to include patients with advanced DMD. “Deramiocel, a heart-derived cellular therapy consisting of human allogeneic cardiosp…
Deramiocel cell therapy slows muscle and heart damage in advanced DMD
A cell therapy called deramiocel could slow muscle weakening in boys and young men with advanced Duchenne muscular dystrophy (DMD), and may also slow heart damage in those who already have heart muscle disease, a phase 3 clinical trial published in The Lancet has found.
The FDA plans to discuss the new therapy today in order to decide whether or not to approve it by the end of August.
LIVE NOW: FDA Cellular, Tissue, and Gene Therapies Advisory Committee Meets on Biologics License for Deramiocel
The FDA Cellular, Tissue, and Gene Therapies Advisory Committee meets at 9:30 a.m. ET on July 29 to discuss and make recommendations on Biologics License Application 125842 from Capricor, Inc. for Deramiocel (human allogeneic cardiosphere-derived cells) in the treatment of cardiomyopathy in Duchenne muscular dystrophy. ...
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