FDA grants priority review to deramiocel for DMD heart disease
Decision expected by Aug. 31 on Capricor's cell therapy approval request
The U.S. Food and Drug Administration (FDA) has accepted Capricor Therapeutics’ application seeking approval of deramiocel — a cell therapy developed by the biotech company to treat heart muscle disease in people with Duchenne muscular dystrophy (DMD) — and granted it priority review that will speed the agency’s decision.
Priority review, which shortens the review period from the standard 10 months to six, is given to therapies with the potential to provide significant benefits in treating serious conditions. A final decision on the biologics license application (BLA), which was submitted in January, is now expected by Aug. 31.
According to Capricor, no potential review issues have been identified by the FDA thus far.
“We are thrilled to announce the acceptance of our BLA bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies,” Linda Marbán, PhD, Capricor’s CEO, said in a company press release. “If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape.”
Deramiocel shown to improve heart, arm function in trial
Duchenne MD is caused by mutations in the DMD gene that result in virtually no dystrophin — a protein that helps protect muscle cells from damage during movements — in the body. This leads to progressive muscle damage that drives the disease’s symptoms.
Besides causing motor issues, DMD also affects the heart muscle, which may result in cardiomyopathy, a condition in which the heart muscle becomes weakened over time, impairing the heart’s ability to pump blood.
Deramiocel (CAP-1002) is comprised of cardiosphere-derived cells, or immature heart cells derived from a healthy donor. These cells produce signaling molecules that induce muscle regeneration and prevent the formation of scar tissue, as well as helping to modulate the immune system.
The BLA submission was primarily based on data from the Phase 2 HOPE-2 trial (NCT03406780) and the HOPE-2 open-label extension (NCT04428476). HOPE-2 enrolled boys and young men with relatively advanced DMD, who were randomly assigned to receive infusions of deramiocel or a placebo every three months, for one year.
The results demonstrated that the cell therapy improved heart and arm function relative to the placebo. Also, the treatment was found to be generally safe and well tolerated, with allergic reactions as the only serious adverse events, which were managed with a common preinfusion treatment.
Further, three-year data from the open-label extension suggested that the therapy continued to show benefits in arm and heart function.
Based on the totality of the safety and efficacy data deramiocel has shown, this potential approval offers patients a first-in-class therapeutic for [heart muscle disease in DMD].
“Deramiocel has shown in multiple clinical trials attenuation of DMD-cardiomyopathy, which is currently one of the leading causes of death in those with DMD,” said Craig McDonald, MD, principal investigator in HOPE-2 and a professor at the University of California, Davis. “Based on the totality of the safety and efficacy data deramiocel has shown, this potential approval offers patients a first-in-class therapeutic for DMD-cardiomyopathy.”
The cell therapy also is being tested in the Phase 3 HOPE-3 trial (NCT05126758). That study enrolled participants ages 10 and older, who were randomly assigned to receive the cell therapy or a placebo every three months for a year. Its main goal is to assess how deramiocel affects arm function.
The FDA had previously granted deramiocel regenerative medicine advanced therapy status, as well as orphan drug and rare pediatric disease designations. The latter two designations are intended to accelerate the development of treatments for rare diseases, defined in the U.S. affecting fewer than 200,000 people.
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