Capricor now seeking FDA approval of DMD therapy deramiocel
Developer starts rolling submission process for US approval
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Capricor Therapeutics has started its rolling submission seeking U.S. approval of deramiocel, a cell therapy candidate to treat cardiomyopathy, a disease of the heart muscle, in people with Duchenne muscular dystrophy (DMD), the company announced.
Rolling submission means that Capricor will be submitting parts of the process to the U.S. Food and Drug Administration (FDA) as they are ready — instead of altogether once completed, as is normally done — with the filing planned to be completed by the year’s end.
The application may be eligible for priority review by the regulatory agency. According to Capricor, this is because deramiocel could potentially provide significant benefits for people with DMD and cardiomyopathy, for whom there are no treatments currently available. Priority review shortens the consideration period to six months from 10.
“This announcement marks an important step in the U.S. regulatory process towards a potential … approval of deramiocel for the treatment of DMD,” Linda Marbán, PhD, CEO of Capricor, said in a company press release.
“An approval of deramiocel would allow us to expedite the delivery of this novel, first-in-class treatment to patients in need,” Marbán said. “We look forward to working with the FDA during this process.”
Application for DMD therapy approval based on HOPE-2 trial data
Duchenne MD is caused by mutations in the DMD gene that result in a lack of functional dystrophin — a protein that helps to protect muscle cells from damage during movements. This leads to progressive muscle weakness and wasting. Along with problems with motor function and development, DMD may also affect the heart muscle.
Deramiocel, formerly called CAP-1002, contains cardiosphere-derived cells that come from a healthy donor. These immature heart cells produce signaling molecules that modulate the immune system, prevent the accumulation of scar tissue, and induce regeneration.
The therapy is delivered directly to the bloodstream once every three months, and is expected to improve the function of the heart and skeletal muscles — those responsible for voluntary movements.
The regulatory application is based on data from the Phase 2 HOPE-2 trial (NCT03406780) and its ongoing open-label extension (NCT04428476), which enrolled boys and young men, 10 and older, with advanced DMD. Most of the participants were unable to walk.
The results from HOPE-2 showed that deramiocel significantly improved heart function and reduced the levels of a heart damage marker compared with a placebo, after one year of treatment. Also, the therapy improved arm function, as a measure of the ability of DMD patients to perform everyday tasks.
After three years of treatment in the extension study, data showed sustained benefits in heart and arm function compared with the decline seen among untreated patients.
Capricor now is sponsoring the Phase 3 HOPE-3 trial (NCT05126758), which is recruiting a similar patient population to that of HOPE-2 at sites in the U.S. The trial is primarily assessing the effects of deramiocel on arm and hand function after a year. Secondary goals are gains in measures of heart health and other functional evaluations.
Deramiocel has received U.S. orphan drug, regenerative medicine advanced therapy, and rare pediatric drug designations for the treatment of DMD. These designations are meant to encourage and accelerate the development of therapies for serious or life-threatening conditions for which there are unmet medical needs.
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