MANY MINDEDAdvisory panel rejects Capricor's heart therapy for Duchenne amid data fight
On July 29, 2026, an FDA advisory committee voted nine to three against the efficacy of Capricor's cell therapy deramiocel for Duchenne muscular dystrophy. The panel considered only the therapy's effect on DMD-associated cardiomyopathy, the specific use Capricor sought to have approved. Deramiocel is made from cardiosphere-derived cells sourced from donor cadavers and given by IV infusion every three months, intended to restore heart and skeletal muscle function.
The meeting turned on a statistical dispute. The resubmitted application drew on new data from the phase 3 HOPE-3 trial, but Capricor CEO Linda Marbán objected to the FDA relying on an analysis plan she called an unfinished, unsigned draft. Capricor's preferred results, published in The Lancet during the meeting, used a later plan version. The company changed its analysis method for a key heart measure the day before data unblinding, and the presented p-value of 0.09 was not statistically significant. Panelists cited the fragility of the results and the extent of the changes.
Duchenne is a fatal rare disease with few options, so a rejection delays access for patients who have little else. That is the cost weighed against the risk of approving a therapy whose benefit is not clearly established.
Advisory votes are recommendations only; the FDA's own decision was expected by August 22 and can override the panel. The FDA and Capricor fundamentally disagree on whether the trial met its endpoints. The acting chair said he would have voted no as a scientist but a qualified yes as a clinician.
Source: Fierce Biotech