Capricor Therapeutics CEO Linda Marbán said on June 26 that the company was surprised to learn the U.S. Food and Drug Administration will hold an advisory committee meeting for its Duchenne muscular dystrophy (DMD) cardiomyopathy cell therapy, deramiocel, before the agency's August target action date.
“We really thought that it’s a very clean data set,” Marbán said from Orlando, where Capricor is presenting five-year data from an ongoing open-label extension study at the Parent Project Muscular Dystrophy conference. “We have not gotten any issues that they want to discuss.”
Marbán suggested possible reasons for the advisory committee meeting could include recent statements by acting FDA Commissioner Kyle Diamantas about increasing adcomms or broader regulatory friction in the DMD space. She pointed to last year's temporary halt of Sarepta Therapeutics' gene therapy Elevidys after patient deaths as a sign regulators may be exercising additional caution. “There’s been so many issues around Duchenne and treatment for Duchenne and Sarepta, maybe they’re being extra careful,” Marbán said.
This is not Capricor's first encounter with unexpected regulatory decisions. In July 2025, deramiocel was rejected by the FDA after then–Center for Biologics Evaluation and Research director Vinay Prasad canceled a scheduled adcomm without warning. The FDA cited insufficient evidence of effectiveness in its complete response letter. However, in December 2025, deramiocel met both primary and secondary endpoints in a pivotal Phase 3 trial: statistically significant benefits in upper-limb function and slowed decline in cardiac function.
The use of externally controlled studies was identified as one major issue during previous review cycles, according to Marbán. Recent changes at the FDA suggest more openness toward such evidence; uniQure and REGENXBIO have recently been allowed to resubmit applications based on externally controlled data following initial discouragement or rejection.
While acknowledging uncertainty about specific topics to be discussed at July’s adcomm, Marbán remained confident about deramiocel’s prospects: “We have a randomized double-blind placebo-controlled trial. We hit the primary endpoint, we hit the secondary endpoints,” she said. “I don’t know what their issues would be.”
