FDA advisory committee meeting for deramiocel in DMD set for July
A decision regarding the therapy's approval is expected by Aug. 31
The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD).
The FDA has informed Capricor Therapeutics the meeting should happen July 30, though Capricor said that date hasn’t been confirmed. At an advisory committee meeting, the FDA gathers independent experts together to offer their opinions on the available data.
“With the FDA advisory committee meeting now scheduled, we look forward to the opportunity to present the totality of evidence supporting the approval of deramiocel for the treatment of Duchenne muscular dystrophy,” Linda Marbán, PhD, CEO of Capricor, said in a company press release. A decision is expected by Aug. 31.
According to the company, the FDA’s application is on schedule. Capricor recently completed a mid-cycle meeting with the agency where no major issues were raised. The FDA also completed its pre-license inspection of Capricor’s facilities where “several observations” were made, but none necessitated changes to its facility or manufacturing setup, according to the company.
“This inspection outcome is a major regulatory milestone, particularly in a field where standards are exceptionally high. It reflects the strength of our manufacturing capabilities and positions us well as we advance toward potential approval,” Marbán said. “With all key review activities progressing on track, we remain focused on delivering this much-needed therapy to the Duchenne community.”
In DMD, gene mutations result in virtually no produced dystrophin, a protein that functions like a shock absorber in muscle cells to prevent cell damage when muscles contract. The absence of functional dystrophin leads to more wear and tear over time, driving muscle weakness and wasting.
DMD affects skeletal muscles, the ones that enable movement, but also has an impact on muscles in the heart, which can lead to cardiomyopathy, or heart muscle disease, a leading cause of death for people with DMD.
How does deramiocel work in Duchenne MD?
Deramiocel, formerly CAP-1002, contains immature heart cells called cardiosphere-derived cells that secrete signaling molecules that should reduce inflammation and fibrosis, and stimulate tissue regeneration.
Capricor sought dermiocel’s approval based on data from a Phase 2 study called HOPE-2 (NCT03406780) and its open-label extension study (NCT04428476). HOPE-2 tested deramiocel against a placebo in 20 boys and young men with DMD, with results indicating deramiocel improved arm and heart function. Long-term data from the extension study have consistently indicated the therapy may help with heart function and preserve arm function.
Capricor recently announced four-year follow-up data from the extension study that indicated that left ventricular ejection fraction (LVEF) — a measure of how well the heart pumps oxygen-rich blood out to the body — decreased by a median of 0.5 points, showing heart function was largely stable. Even greater benefits were observed in patients with a starting LVEF higher than 45%, according to Capricor, which suggests early treatment may lead to better heart function stabilization.
Scores on the Performance of the Upper Limb (PUL), a measure of arm and hand function, worsened by 0.6 points on average in the fourth year of follow-up. By comparison, in the first year of the study, PUL scores worsened by 1.8 points. This suggests long-term therapy with deramiocel is slowing the decline in arm function for DMD patients, Capricor maintains.
“These four-year data reinforce the strength and durability of deramiocel’s clinical benefit and favorable safety profile across both cardiac and skeletal muscle function … We thank the patients, families, and clinicians who have been instrumental in advancing this program” Marbán said in a separate press release.
The four-year data were presented at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference this month in Las Vegas.
“Cardiomyopathy remains one of the leading causes of mortality in Duchenne and addressing this aspect of the disease is critical to improving outcomes,” said Pat Furlong, founding president and CEO of PPMD. “The long-term data from the HOPE-2 [open-label extension] study are encouraging, particularly in demonstrating cardiac stabilization over four years. Deramiocel represents an important therapeutic approach and we support continued progress through the regulatory pathway to ensure that treatments targeting both heart and muscle function are available to our community as quickly as possible.”
Deramiocel is also being developed for Becker muscular dystrophy. The FDA has granted it orphan drug status, which confers incentives for drug development such as seven years of market exclusivity if approved, for this type of muscular dystrophy.
“We believe Deramiocel holds promise for patients with Becker, particularly given the overlap in disease pathology [processes] with Duchenne,” Marbán said in another press release. “Deramiocel has demonstrated potential to treat the serious cardiac and skeletal muscle complications of Duchenne muscular dystrophy and, based on the overlap in disease pathology, may also offer benefit to patients with Becker muscular dystrophy.”
About the Author
