Deramiocel (CAP-1002)

Development notes

HOPE-2/HOPE-3 trials. 3/4/25- BLA accepted & priority review. PDUFA date August 31, 2025. 7/11/25- CRL received. 9/9/25- Responded to FDA CRL posting. 9/25/25- HOPE-3 trial completed. 12/3/25– HOPE-3 met primary and key cardiac endpoints. 01/20/26:- FDA requested HOPE-3 CSR; company plans BLA resubmission in February 2026 to enable new PDUFA. 02/24/26:- HOPE-3 CSR submitted; FDA BLA review ongoing. Detailed HOPE-3 results to be presented at MDA on March 11, 2026. 03/10/26:- FDA resumed BLA review; Class 2 resubmission. PDUFA action date set for August 22, 2026. 03/12/26:- HOPE-3 late-breaking MDA 2026 data show significant functional and cardiac benefits; BLA review ongoing, PDUFA August 22, 2026. 04/22/26:- HOPE-3 Phase 3 data presented at AAN 2026. PDUFA action date remains August 22, 2026. 05/12/26:- PDUFA August 22, 2026 confirmed; labeling talks expected soon; HOPE-3 data strengthened cardiac and functional outcomes. 06/26/26:- Five-year HOPE-2 OLE data presented at PPMD June 27. PDUFA action date remains August 22, 2026. 07/27/26:- FDA Adcomm upcoming for Deramiocel BLA; company defends HOPE-3 analysis. PDUFA Aug 22, 2026. 07/30/26:- Adcomm voted 9-3 against cardiomyopathy effectiveness; PDUFA remains August 22, 2026. 08/13/26:- HOPE-3 results were published; BLA remains under FDA review. PDUFA decision will occur August 22, 2026.