FDA Advisory Committee Votes 9-3 Against Capricor's Deramiocel in DMD Cardiomyopathy
In a major clinical and regulatory setback for Capricor Therapeutics, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3 on July 29, 2026, against finding sufficient evidence supporting the effectiveness of deramiocel (CAP-1002). The allogeneic cardiosphere-derived cell therapy was being reviewed for the treatment of cardiomyopathy in male patients with Duchenne muscular dystrophy (DMD).
The negative vote came just days after the FDA released highly critical briefing documents on July 27, 2026, which triggered a massive stock plunge of over 64% for Capricor and subsequent shareholder class-action lawsuits.
Misalignment on Statistical Analysis and Indication
The high-stakes AdCom meeting devolved into what observers described as "chaotic" statistical confusion, centered explicitly on disputes over the statistical analysis plan (SAP) and post-hoc endpoints.
According to Capricor CEO Linda Marbán, the company was stunned by the FDA’s posture and reviewer arguments. The biotech had entered the meeting believing it possessed a clean, pivotal Phase 3 dataset from the HOPE-3 trial. However, the FDA reviewers raised severe concerns regarding the integrity of the data collection and argued that the benefits shown on skeletal muscle function did not translate into evaluable, robust evidence of effectiveness for cardiomyopathy.
Furthermore, Capricor and the FDA were misaligned on:
- Statistical Analysis Plan (SAP): Disagreements over which version of the SAP (including version 1.1) should be reviewed.
- Indication: The question put before the committee addressed a narrower cardiomyopathy indication than Capricor had originally applied for, which did not align with the Phase 3 trial's primary endpoint.
- CMC Deficiencies: Chemistry, Manufacturing, and Controls deficiencies cited in a prior Complete Response Letter (CRL) remained unresolved, presenting an ongoing liability.
Marbán has refused to rule out legal action against the FDA over how the statistical analysis was framed and presented to the committee.
Looming August PDUFA Verdict
The FDA is not bound by the advisory committee’s vote, but typically follows its recommendations. The negative AdCom vote significantly clouds deramiocel’s regulatory path as the FDA approaches its August 22, 2026 PDUFA target action date.
Industry analysts widely expect the FDA to reject the Biologics License Application (BLA) and issue a third Complete Response Letter (CRL), arguing that the clinical results are insufficient to support accelerated approval. This outcome highlights the persistent regulatory hurdles cell therapies face under the FDA's current statistical and clinical standards for rare diseases.
"It was very clear from the voting question and sort of how [FDA] pitched it with stat plan 1.1 that they seem to have some type of ulterior motive in trying to take this therapeutic down, and I can't figure out why," Linda Marbán, Ph.D., CEO of Capricor, told BioSpace.
"The CTGTAC vote against deramiocel centered explicitly on disputes over statistical analysis and post-hoc endpoints. The committee’s concern was not that the therapy showed no signal, but that the sponsor's briefing document and the FDA's review memorandum were not aligned on the analytical hierarchy," reported Clinical Trial Vanguard.