Deramiocel (CAP-1002)

Deramiocel remains an investigational treatment. Following a complete response letter and resubmission, the sponsor reported a PDUFA target action date of November 22, 2026 and a refined proposed indication focused on upper limb function. The advisory committee voted 3 in favour and 9 against effectiveness for DMD-associated cardiomyopathy, with no abstentions; the vote was non-binding. The published upper-limb analysis, the nonsignificant key cardiac endpoint, and FDA’s assessment of the prespecified analyses leave uncertainty about the efficacy conclusions and the eventual indication. A target action date does not establish approval.