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2 clusters · 10 sources · 3 days · First seen 3 days ago · Last updated about 19 hours ago

Categories: HEALTH

Deramiocel DMD trial & FDA panel rejection

Entities: Linda Marban · Food and Drug Administration · Capricor Therapeutics · deramiocel · Duchenne muscular dystrophy

Overview

The FDA advisory committee voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ experimental stem‑cell therapy deramiocel for Duchenne muscular dystrophy‑related cardiomyopathy, reinforcing earlier staff concerns about insufficient efficacy evidence and post‑hoc changes to key outcome analyses. The Phase III HOPE‑3 trial, which enrolled 106 boys and young men (54 receiving deramiocel, 52 receiving placebo), showed a 54 % slower decline in arm function and up to a 91 % slower progression of heart‑function loss in participants with pre‑existing cardiomyopathy. Allergic‑type reactions were more frequent (42 % vs 15 % placebo) but remained mild to moderate, and no deaths occurred. The panel also questioned the baseline cardiac function of participants. The vote means a final FDA decision is still expected by 22 August, and Capricor’s shares fell more than 66 % after the staff briefing. CEO Linda Marbán called the outcome a setback while emphasizing deramiocel’s potential as the first therapy to directly target heart disease in Duchenne patients.

Claims

What the coverage asserts, and how well corroborated each claim is across sources.

[● 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)
[● 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] 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] The FDA is expected to issue a final decision on deramiocel by August 22. (2024-07-29)
[● 3 SOURCES] Capricor’s shares fell more than 66 % after FDA staff concerns were published. (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)
[● 2 SOURCES] Allergic‑type reactions occurred in 42 % of deramiocel recipients versus 15 % of placebo; no deaths were reported. (2024-07-29)

Timeline

about 19 hours ago

[HEALTH] 8 sources
Capricor Therapeutics' deramiocel fails FDA advisory panel vote despite trial benefits
FDA advisory panel voted 9‑3 against Capricor's deramiocel for Duchenne cardiomyopathy, citing insufficient evidence; shares fell >66%. A phase‑III trial showed the drug slowed arm decline by 54 % and heart‑d病
3 days ago

[HEALTH] 2 sources
Capricor Therapeutics’ Deramiocel Fails FDA Phase 3 Trial in Duchenne Muscular Dystrophy
The FDA concluded Capricor Therapeutics’ Phase 3 trial of the stem‑cell therapy deramiocel missed primary and secondary endpoints for Duchenne muscular dystrophy, citing insufficient efficacy and safety issues,

Sources

archynetys.com · archynewsy.com · expansion.com · it-boltwise.de · kfgo.com · news-medical.net · newswise.com · proiezionidiborsa.it · wixx.com · wvfm1065.com

This summary has been updated 4 times: see revision history