Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy was not recommended for approval by an FDA advisory committee on July 29, with the company’s CEO voicing frustration over the agency’s review process. The advisory committee voted 9-3 against recommending approval of deramiocel, citing concerns about the evidence supporting its effectiveness.
Capricor CEO Linda Marbán addressed the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee, saying parts of the FDA’s briefing document were difficult to reconcile with actual events. "When I first read the FDA’s briefing document, parts of it were so hard to reconcile with what actually occurred," Marbán said during her presentation. Despite her remarks and testimony from families affected by Duchenne muscular dystrophy (DMD), most committee members remained unconvinced. Janet Turk Wittes, a member of the committee and expert in randomized controlled trials, said, "The word of the day seems to be fragile. The results were very fragile... I simply didn’t see enough evidence of benefit to vote yes." Cynthia Tifft also commented on her decision: "Given the question that was asked, I think the evidence is really not that compelling... So I get it," she said.
Some members supported deramiocel. Patient representative Christopher Mariano Cassidy Jr. criticized how technicalities in statistical analysis led to data being dismissed: "So much of [the FDA’s reliability report] is devoted to identifying technicalities and procedural errors and using them as a basis for dismissing entire data sets out of hand without even examining." Another advocate, Debra Dunn, voted yes but expressed uncertainty: “Am I 100% vested in that? I don’t know ... listening to the patients and to their families. I’m a mom. I have a heart. They don’t have an alternative.”
The rejection follows previous setbacks for Capricor; last July, deramiocel was rejected when Phase 2 data did not meet requirements for substantial evidence of effectiveness according to regulatory standards at that time. In December 2025, Capricor reported positive results from its pivotal Phase III HOPE-3 trial but briefing documents released before this week’s meeting stated there was no statistically significant difference between deramiocel and placebo at 12 months.
A major point of contention involved which version of a statistical analysis plan should guide evaluation—Capricor favored SAP version 3.0 finalized before unblinding while FDA used SAP version 1.1, which they considered current at study initiation; both sides disagreed on whether proper procedures had been followed regarding changes in analysis plans.
Marbán concluded by highlighting consistent positive trends across all clinical studies conducted by Capricor over ten years: “The most important takeaway from our decade of clinical work in Duchenne muscular dystrophy is that every study points in the same positive direction across both skeletal and cardiac function,” she said.
