FDA States Advisory Committee Meeting Will be Held for Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel

In a mid-cycle review meeting between the FDA and Capricor Therapeutics regarding the latter’s biologics license application (BLA) for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, the agency stated its intent to hold an advisory committee meeting regarding the BLA.¹

Alongside this, however, the FDA noted in the meeting that the review committee had found no major deficiencies with the BLA package. The date for the advisory committee meeting has not yet been determined.

“The successful completion of our mid-cycle review meeting along with the upcoming advisory committee meeting represents major milestones on the path towards approval of deramiocel,” Linda Marbán, PhD, thechief executive officer of Capricor, said in a statement.¹ “Deramiocel is a first-in-class cellular therapy with the potential to halt or slow the progression of DMD-cardiomyopathy, and we are pleased to have the opportunity to present the efficacy and safety data to the advisory committee. We have been actively preparing for an advisory committee meeting, and we look forward to providing the physician and patient perspectives to highlight the weight of evidence supporting the transformative potential of deramiocel in treating DMD-cardiomyopathy.”

The BLA for deramiocel was accepted by the FDA with priority review in March 2025.² The Prescription Drug User Fee Act (PDUFA) action date for the BLA was set as August 31, 2025, and in the update regarding the mid-cycle review meeting the FDA confirmed that the package remains on track for this date.^1,2 The BLA is supported by results from the completed HOPE-2 clinical trial (NCT03406780) and HOPE-2’s ongoing open-label extension (OLE) study (HOPE-2-OLE; NCT04428476), with data from these studies being compared to an FDA funded natural history dataset. Notably, an ongoing phase 3 clinical trial dubbed HOPE-3 (NCT05126758) is also evaluating Deramiocel in DMD, although efficacy data from this study is not part of the review package for the BLA. According to the clinicaltrials.gov page for HOPE-3, which was most recently updated on February 4, 2025, the study is active, with an actual enrollment of 104 patients, and no longer recruiting new participants.

Notably, data from HOPE-2-OLE were recently presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19, in Dallas, Texas. In an interview with CGTLive® after the conference, Marbán explained the mechanism behind Deramiocel and how it is intended to address unmet needs in DMD, and then went over the key findings that were reported at the conference.

“What we were able to see is that year over year, again, we are showing improvement in cardiac function compared to natural history,” Marbán told CGTLive. “One could argue that the Performance of the Upper Limb or the North Star Ambulatory Assessment, or any of these others are volitional: you know you're getting a drug so you might perform better. You cannot wish your heart better. It just does what it does. And so the fact that we have this objective measure of MRI, both in our treated patients and in the natural history patients, and we see a 6.9% improvement over 24 months in our treated patients compared to the standard of care guys who are getting the best cardiac meds have to offer, as are our patients, we now have a really strong metric of improvement. Skeletal muscle function improvement and cardiac muscle function improvement leads to a really great opportunity with a very safe therapeutic in deramiocel.”

REFERENCES
1. Capricor Therapeutics announces completion of mid-cycle review meeting with FDA on Deramiocel for the treatment of Duchenne muscular dystrophy cardiomyopathy. News release.Capricor Therapeutics. May 5, 2025. Accessed May 9, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/311/capricor-therapeutics-announces-completion-of-mid-cycle
2. Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy. News release. Capricor Therapeutics. March 4, 2025. Accessed March 4, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority