A US Food and Drug Administration (FDA) advisory committee meeting (AdCom) has voted against Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy (DMD), capping an extraordinary meeting in which the agency and biotech fundamentally disagreed on how key study data was analysed.
In a meeting which saw a buildup dominated by disagreements over the FDA’s briefing document, committee members voted 9-3 against recommending approval of Capricor’s deramiocel. The panel will now pass their findings to the FDA’s review team. While the agency is not beholden to the outcome of AdComs, it often follows their recommendation.
Capricor’s application is specifically for DMD-associated cardiomyopathy and the preservation of skeletal muscle function. However, the panel was asked by Capricor to consider deramiocel’s full potential in the disease.
The FDA already rejected the therapy once on the basis of a Phase II trial, issuing a complete response letter (CRL) citing lack of substantial effectiveness in July 2025. Capricor then issued a public statement refuting the claims.
Capricor came back to the regulatory table with a pivotal study and an updated biologics license application (BLA). Conversation at the AdCom meeting centred on Capricor’s Phase III HOPE-3 trial. In December 2025, the biotech announced the study had achieved both primary and secondary endpoints, sending its stock up significantly.
However, in briefing documents, the FDA claimed that HOPE-3 in fact missed its efficacy endpoints, contending that deramiocel was no better than placebo.
After the briefing documents were published, Capricor’s stock dropped significantly, by just over 70%, from a 24 July close of $19.83 to a 27 July open of $5.87. The company’s stock has risen slightly since then, closing at $6.57 on 29 July after the AdCom vote.
Capricor’s CEO Linda Marbán tackled the briefing documents head on when opening her company’s presentation at the meeting.
“When I first read the FDA's briefing document, parts of it were so hard to reconcile with what actually occurred,” she said in front of committee members.
Marbán went on to criticise aspects of the FDA’s evaluation, specifically using an unfinished statistical analysis plan to perform numerous analyses.
“This would be like your professor grading your term paper on an early draft you have never even submitted,” she alleged.
Results from the study, using a later SAP, were published in The Lancet during the meeting. Capicor said in a separate statement that “independent peer review provides external validation of the trial's design, statistical methodology and findings”.
However, committee members were not convinced. The experts argued Capricor changed important efficacy analyses after the trial finished and debated whether cardiac measurements convincingly demonstrated meaningful patient benefit. Some members were also concerned about potential increases in left ventricular volume – a hallmark of disease progression. Overall, data fragility was the main theme for members who voted against the therapy.
“We have markedly fragile results that can be changed with different imputation strategies [and] different inclusions of different people,” commented Dr John Teerlink, professor and cardiologist.
“That was how I’ve been feeling, that these data are very fragile. They’re dependent on one or two people,” said Janet Turk Wittes, a biostatistician on the panel.
When the meeting opened for public comment, patients, patient advocates and doctors spoke about deramiocel’s positive effects and helping patients maintain dignity and gain independence.
Deramiocel is comprised of cardiosphere-derived cells (CDCs) derived from donor heart tissue and then administered into patients via an intravenous injection.
Capricor did not immediately respond to Pharmaceutical Technology’s request for comment on the outcome of the AdCom meeting.
