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FDA panel rejects Capricor's deramiocel therapy for Duchenne heart disease
On July 29, the U.S. Food and Drug Administration’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ cell therapy deramiocel for cardiomyopathy in Duchenne muscular dystrophy. The panel cited insufficient evidence of efficacy and concerns that the company had altered key statistical analyses after completing its late‑stage study. FDA staff had previously raised the same issues. The advisory vote is non‑binding, but the agency is expected to render a final decision by August 22, and analysts predict another Complete Response Letter rejecting the therapy.
The decision triggered a sharp market reaction: Capricor shares fell nearly 60% in pre‑market trading and have dropped more than 66% since the staff concerns were published, wiping out roughly two‑thirds of the company’s market value. The therapy’s clinical data come from the phase III HOPE‑3 trial, which enrolled 106 male participants aged 10 to 22 with advanced Duchenne muscular dystrophy—54 received deramiocel and 52 received placebo. The trial met its primary endpoint, showing a 54 % slower decline in upper‑limb function compared with placebo. The overall cardiac primary endpoint (change in left‑ventricular ejection fraction) did not reach statistical significance, though a prespecified subgroup with documented cardiomyopathy showed a nominally significant benefit. Safety findings indicated allergic‑type reactions in 42 % of treated patients versus 15 % of placebo recipients, with no deaths reported.
Entities
Capricor Therapeutics · Duchenne muscular dystrophy · Food and Drug Administration · HOPE-3 trial · HOPE‑3 trial · Linda Marban · National Institutes of Health · The Lancet · U.S. Food and Drug Administration · United States · deramiocel
Claims
What the coverage asserts, and how many sources carry each claim.
- [● 4 SOURCES] FDA staff raised concerns that Capricor had not provided sufficient evidence of effectiveness and had changed key analyses after the late‑stage study. jack1065.com · wixx.com · kfgo.com · wdez.com
- [● 5 SOURCES] Capricor shares fell nearly 60% in pre‑market trading and more than 66% since FDA staff concerns were published. wixx.com · kfgo.com · jack1065.com · wdez.com · kelo.com
- [● 4 SOURCES] Deramiocel participants lost arm function about 54 % more slowly than placebo participants. www.insideprecisionmedicine.com · www.news-medical.net · www.newswise.com · www.expansion.com
- [● 3 SOURCES] The FDA advisory panel voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ deramiocel therapy for Duchenne‑related cardiomyopathy. jack1065.com · wixx.com · kfgo.com
- [● 3 SOURCES] In participants with existing cardiomyopathy, deramiocel preserved heart function better than placebo, with up to a 91 % slower progression of cardiac decline. www.news-medical.net · www.expansion.com · www.newswise.com
- [● 4 SOURCES] The FDA is expected to issue a final decision on deramiocel by August 22. wixx.com · kfgo.com · jack1065.com · wdez.com
- [● 2 SOURCES] Allergic‑type reactions occurred in 42 % of deramiocel recipients versus 15 % of placebo; no deaths were reported. www.news-medical.net · www.newswise.com
- [● 4 SOURCES] The HOPE‑3 phase III trial enrolled 106 male participants aged 10‑22, with 54 receiving deramiocel and 52 receiving placebo. www.insideprecisionmedicine.com · www.news-medical.net · www.newswise.com · www.expansion.com
- [● 4 SOURCES] The FDA advisory committee voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ deramiocel therapy for Duchenne‑related cardiomyopathy. jack1065.com · wixx.com · kfgo.com · wdez.com
- [● 4 SOURCES] The HOPE‑3 phase III trial enrolled 106 male participants aged 10‑22 with advanced Duchenne muscular dystrophy. www.insideprecisionmedicine.com · www.news-medical.net · www.expansion.com · www.newswise.com
- [● 2 SOURCES] In participants with cardiomyopathy, deramiocel showed a nominally significant benefit in left‑ventricular ejection fraction. www.insideprecisionmedicine.com · jack1065.com