Upcoming Capricor, FDA meeting to focus on DMD treatment deramiocel
Agency, company to discuss plans for cell therapy's approval application
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Capricor Therapeutics will soon meet with the U.S. Food and Drug Administration (FDA) to discuss plans for an application seeking approval of the company’s CAP-1002 cell therapy, now named deramiocel, as a treatment for Duchenne muscular dystrophy (DMD).
The FDA has scheduled the pre-biologics license application (BLA) meeting for the third quarter of this year, according to a company press release. At the meeting, Capricor and the FDA will discuss exactly what needs to go into the application for deramiocel — and will outline the timeline for getting the application submitted and reviewed.
“We continue to move rapidly towards potential approval of deramiocel for the treatment of DMD,” said Linda Marbán, PhD, CEO of Capricor . Marbán added that the meeting “will finalize our BLA filing plans and discuss available options to expedite the filing of our BLA.”
BLA approval is needed to market a new drug in the U.S. The scheduling of the pre-BLA meeting comes just after the completion of a type-B meeting between Capricor and the FDA, which the company deemed “successful.” At that meeting, the FDA agreed to host the more in-depth pre-BLA meeting.
The regulatory agency also signaled that Capricor may submit a rolling submission for deramiocel — meaning the company will be able to give the FDA each part of the application for review as soon as the section is ready, instead of having to wait until the whole application is completed before submitting, as is typically done.
Rolling applications can help speed the application process, but because the FDA has limited resources to work with, it only allows rolling applications for experimental treatments that have the potential to fill major unmet medical needs.
“We recognize the FDA’s willingness to bring impactful therapies to market as quickly as possible due to the enormous unmet needs of patients with DMD,” Marbán said.
Deramiocel aims to promote heart health in Duchenne patients
Deramiocel is a cell therapy that contains cardiosphere-derived cells, or immature heart cells that are able to release signaling molecules that promote heart health. In patients with Duchenne muscular dystrophy, the most common type of the disease, the therapy is expected to improve muscle strength, with an aim of helping cardiac health. It is given via infusions into the bloodstream every three months.
Capricor expects that the cell therapy may be used to complement other treatment approaches such as Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy that is now authorized in the U.S. for Duchenne patients ages 4 and older.
“While we are delighted with the approval of gene therapy for DMD, we believe that it will take multiple therapies to combat DMD effectively,” Marbán said.
“Based on the need to address the secondary consequences of DMD, we believe deramiocel can serve as a potential anchor therapy for DMD patients,” Marbán added.
While we are delighted with the approval of gene therapy for DMD, we believe that it will take multiple therapies to combat DMD effectively. … Based on the need to address the secondary consequences of DMD, we believe deramiocel can serve as a potential anchor therapy for DMD patients.
A Phase 3 clinical trial called HOPE-3 (NCT05126758), sponsored by Capricor, is now testing one year of treatment with deramiocel versus a placebo in boys and young men with DMD. Top-line results are expected later this year.
The company also is continuing to collect long-term data from the open-label extension (NCT04428476) of the Phase 2 HOPE-2 trial (NCT03406780). New data from HOPE-2, shared by the Capricor earlier this month, indicated that deramiocel helped to slow arm function decline and stabilize heart health after up to three years of treatment.
Three-year data from HOPE-2 will be discussed this weekend at the Parent Project Muscular Dystrophy (PPMD) annual conference, taking place in Orlando, Florida.
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