Capricor’s June 26 press release put the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee on the calendar for July 29, less than four weeks before the Aug. 22 PDUFA date. CEO Linda Marbán called it a “surprise.” That’s an understatement.
In June 2025, deramiocel’s original adcomm appeared on the Federal Register, then vanished ten minutes later after then-CBER director Vinay Prasad canceled it unilaterally. The complete response letter that followed in July 2025 cited failure to meet the statutory requirement for “substantial evidence of effectiveness” and the externally controlled study design as deficiencies.
The Phase 3 HOPE-3 trial changed the picture. The 106-participant randomized controlled trial, reported December 3, 2025, showed deramiocel slowing disease progression 54% on the PUL v2.0 primary endpoint (p=0.029) and cardiac decline by 91% on LVEF. Capricor’s Class 2 resubmission cleared in March 2026 with no flagged review issues; the FDA set an Aug. 22 PDUFA date.
Marbán offered two theories for the adcomm. Acting Commissioner Kyle Diamantas publicly said he wants more advisory committees. And the DMD space carries friction: after Sarepta halted Elevidys shipments following two patient deaths, “maybe they’re being extra careful,” she said.
FDA called adcomms for Capricor and Replimune on the same day, and former regulators at BIO cited the revival as a return to normalcy after Prasad’s nine-month advisory committee drought. That benefits rare-disease sponsors who’ve had no way to read agency risk tolerance on external controls. Marbán doesn’t buy the clean-house narrative yet — but says she isn’t worried about the BLA.
The question list hasn’t arrived. Watch July 29.
Rebecca Lauren