FDA set to decide on deramiocel for DMD cardiomyopathy
Agency found no issues with Capricor application; on track for Aug. 31 decision
The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August.
The FDA agreed this year to review an application to approve deramiocel for DMD cardiomyopathy, or disease of the heart muscle. The agency granted the application priority review, shortening the review time to six months down from the usual 10 months, with a decision expected by Aug. 31.
Deramiocel’s developer Capricor Therapeutics has now completed a mid-cycle review meeting with the FDA. According to Capricor, the FDA didn’t identify any significant issues and is on track for a decision.
“The successful completion of our mid-cycle review meeting along with the upcoming advisory committee meeting represents major milestones on the path towards approval of deramiocel,” Linda Marbán, PhD, Capricor’s CEO, said in a company press release.
The FDA has indicated that it plans to hold an advisory committee meeting for the application where it will convene a group of outside experts to provide their opinions on the therapy’s available data.
“We have been actively preparing for an advisory committee meeting, and we look forward to providing the physician and patient perspectives to highlight the weight of evidence supporting the transformative potential of deramiocel in treating DMD cardiomyopathy,” Marbán said.
How does deramiocel work in DMD cardiomyopathy?
DMD is caused by mutations that impair the production of dystrophin, a protein that helps maintain muscle cell health. The disease is marked by weakness and wasting of skeletal muscles, that is, the muscles that move the body around, and it also causes damage to heart muscles. This often leads to cardiomyopathy, which is a leading cause of mortality in DMD.
Deramiocel, formerly CAP-1002, contains immature heart cells called cardiosphere-derived cells, which secrete signaling molecules that are expected to limit inflammation and boost blood vessel growth to promote muscle regeneration.
“Deramiocel is a first-in-class cellular therapy with the potential to halt or slow the progression of DMD cardiomyopathy, and we are pleased to have the opportunity to present the efficacy and safety data to the advisory committee,” Marbán said.
Capricor’s application for deramiocel is based on data from the Phase 2 HOPE-2 clinical trial (NCT03406780) and its open-label extension study (NCT04428476). HOPE-2 tested deramiocel against a placebo in 20 people with DMD, and its results suggested the therapy improved arm and heart function. Long-term data from the extension study have consistently indicated that deramiocel can aid heart function and preserve arm function.
Capricor is running another clinical trial called HOPE-3 (NCT05126758) that’s further exploring the effects of deramiocel in more than 100 boys and men with DMD. That study, which is not part of the FDA application, is expected to have results later this year.
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