Capricor Therapeutics' deramiocel fails FDA advisory panel vote despite trial benefits

deramiocel Capricor Therapeutics FDA advisory panel Duchenne muscular dystrophy HOPE-3 trial

The U.S. Food and Drug Administration's advisory committee voted 9‑to‑3 against recommending approval of Capricor Therapeutics' cell therapy deramiocel for Duchenne muscular dystrophy‑related cardiomyopathy. FDA staff had raised concerns that the company did not provide sufficient evidence of effectiveness and had altered key analyses after the late‑stage study, questioning both the arm‑function and heart‑function endpoints. The panel’s decision aligns with the agency’s earlier refusal to approve the therapy and the expectation that a final FDA decision will be issued by August 22. Capricor’s shares fell more than 66 % after the staff concerns were made public.

In the phase‑III HOPE‑3 trial, 106 boys and young men aged 10‑22 with advanced Duchenne were randomized to deramiocel (54) or placebo (52). After one year, participants receiving deramiocel lost arm function about 54 % more slowly than placebo, and heart‑function decline was up to 91 % slower in those with existing cardiomyopathy. The therapy was generally safe; allergic‑type reactions occurred in 42 % of deramiocel recipients versus 15 % of placebo, 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 well corroborated each claim is across sources.

[● 3 SOURCES] FDA staff raised concerns that Capricor had not provided sufficient evidence of effectiveness and had changed key analyses after the late‑stage study. (supporting_article_ids)
[● 3 SOURCES] Capricor’s shares fell more than 66 % after FDA staff concerns were published. (2024-07-29)
[● 4 SOURCES] After one year, deramiocel participants lost arm function about 54 % more slowly than placebo participants. (2024-07-29)
[● 3 SOURCES] The FDA advisory panel voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ deramiocel therapy for Duchenne‑related cardiomyopathy. (2024-07-29)
[● 3 SOURCES] In participants with existing cardiomyopathy, deramiocel preserved heart function better than placebo, with up to a 91 % slower progression of cardiac decline. (2024-07-29)
[● 3 SOURCES] The FDA is expected to issue a final decision on deramiocel by August 22. (2024-07-29)
[● 2 SOURCES] Allergic‑type reactions occurred in 42 % of deramiocel recipients versus 15 % of placebo; no deaths were reported. (2024-07-29)
[● 4 SOURCES] The HOPE‑3 phase III trial enrolled 106 male participants aged 10‑22 with advanced Duchenne, 54 receiving deramiocel and 52 receiving placebo. (supporting_article_ids)
[● 3 SOURCES] The FDA advisory committee voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ deramiocel therapy for Duchenne‑related cardiomyopathy. (supporting_article_ids)
[● 4 SOURCES] The HOPE‑3 phase III trial enrolled 106 male participants aged 10‑22 with advanced Duchenne muscular dystrophy. (2024-07-29)