Amid its ongoing review of the cell therapy deramiocel for people with Duchenne muscular dystrophy (DMD), the U.S. Food and Drug Administration (FDA) has scheduled a meeting with Capricor Therapeutics, its developer, to discuss the company’s resubmission of an application seeking the treatment’s approval.
That meeting — between Capricor and the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee, or CTGTAC — will be held on July 29, according to a press release from the developer, which noted that the session will be available for live streaming.
A decision from the agency on whether or not to approve deramiocel for DMD is expected by Aug. 22.
Capricor had previously requested the therapy’s approval, primarily based on Phase 2 clinical trial results, but the FDA last year rejected that application. In its decision, the regulatory agency stated that there was insufficient evidence of the treatment’s effectiveness. Capricor has since worked to bolster its case for approval.
“We are encouraged by the opportunity to bring Deramiocel before the Advisory Committee and engage directly with the FDA, the DMD patient community, and the physicians who care for them,” said Linda Marbán, PhD, Capricor’s CEO, adding that the company is confident in its application. Additional information about the meeting is available on the company’s website, per the release.
DMD is caused by genetic mutations that result in virtually no production of dystrophin, a protein that normally helps protect muscles from damage during movement. This leads to symptoms such as progressive muscle weakness and wasting.
Cardiomyopathy, a condition in which the heart becomes enlarged and thickened, and its ability to pump blood is impaired, is the leading cause of death among people with DMD.
New trial data being used to seek approval of deramiocel for DMD
Deramiocel (CAP-1002) is a cell-based therapy that contains cardiosphere-derived cells — a population of immature heart cells with anti-inflammatory and immunomodulatory properties. It was designed to improve muscle strength and heart health in people with DMD.
The therapy is made from human donor hearts, and is delivered to patients via infusion. Then, inside the body, the therapeutic cells release tiny particles called exosomes that are taken up by immune cells called macrophages to drive them into a healing mode.
A since-completed Phase 2 trial dubbed HOPE-2 trial (NCT03406780) tested deramiocel versus a placebo in 20 people with DMD. The results showed that the treatment improved heart and arm function in boys and young men at advanced stages of the disease. Benefits were also reported at four years of treatment from the HOPE-2 open-label extension (NCT04428476), which overall found largely stable heart function and a slowed decline of arm function among the participants.
The Phase 3 HOPE-3 study (NCT05126758) then enrolled 106 boys and young men with DMD, most of whom were unable to walk (85%) and had cardiomyopathy (75%). Each was randomly assigned to receive infusions of either deramiocel or a placebo every three months for one year.
Top-line data from that trial showed that the therapy stabilized the progression of left ventricular ejection fraction — a measure of how effectively the heart pumps blood out to the body. The effects on heart function were particularly pronounced in patients with cardiomyopathy at the start of the study, who actually experienced slight improvements.
Besides the heart, deramiocel also slowed decline in arm and hand function relative to the placebo, as measured with the Performance of the Upper Limb test. The resubmitted application includes the Phase 3 trial data, according to Capricor.
We have confidence in the totality of evidence supporting Deramiocel, which has demonstrated clinically meaningful, statistically significant skeletal and cardiac benefits with a consistent safety profile, across multiple studies supporting its potential as a first-in-class therapy for Duchenne muscular dystrophy.
“Our focus remains on supporting the Agency’s review and preparing for this meeting, with the urgent needs of the DMD community guiding every step, and we remain committed to bringing this therapy to the families who need it,” Marbán said.
The company noted that deramiocel achieved statistical significance on its primary goal, or endpoint, and secondary endpoints in the Phase 3 trial.
“We have confidence in the totality of evidence supporting Deramiocel, which has demonstrated clinically meaningful, statistically significant skeletal and cardiac benefits with a consistent safety profile, across multiple studies supporting its potential as a first-in-class therapy for Duchenne muscular dystrophy,” Marbán said.
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