Deramiocel (CAP-1002)
Cardiomyopathy associated with Duchenne muscular dystrophy · Patients with Duchenne muscular dystrophy cardiomyopathy
CAPR · Capricor Therapeutics
監管認定
完整時間線
6 個里程碑FDA decision expected
PDUFA target action date for the Class 2 resubmission of the deramiocel BLA.
Capricor — FDA resumed review and established the new PDUFA date↗CTGTAC voted 9–3 against effectiveness
The FDA advisory committee voted 9–3 that the available evidence did not establish deramiocel's effectiveness for cardiomyopathy in Duchenne muscular dystrophy. The recommendation is non-binding and the FDA decision remained pending.
FDA — July 29 CTGTAC meeting materials and webcast↗FDA resumed review of the BLA
FDA accepted the response to the CRL as a complete Class 2 resubmission and assigned an August 22, 2026 target action date.
Capricor — New PDUFA date for the deramiocel resubmission↗HOPE-3 met its primary and key cardiac endpoints
The randomized Phase 3 study met the PUL v2.0 primary endpoint and the LVEF key secondary endpoint, with statistical significance also reported across all Type I error-controlled secondary endpoints.
Capricor — Positive Phase 3 HOPE-3 topline results↗相關新聞
Capricor Therapeutics Reports Second Quarter 2026 Financial Results and Provides Corporate Update
2026年8月10日Capricor Therapeutics to Report Second Quarter 2026 Financial Results and Provide Recent Corporate Update on August 13
2026年7月30日Capricor Therapeutics Provides Update on FDA Advisory Committee Meeting for Deramiocel
2026年7月29日The Lancet Publishes HOPE-3 Data for Capricor Therapeutics’ Deramiocel in Duchenne Muscular Dystrophy
2026年7月27日Capricor Comments on Briefing Materials Ahead of July 29 FDA Advisory Committee Meeting for Deramiocel