Capricor Therapeutics announced the completion of a mid-cycle review meeting with the FDA for its Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA reported no significant deficiencies in the application and confirmed the PDUFA target action date of August 31, 2025, with plans for an advisory committee meeting beforehand. The BLA is based on data from Capricor’s Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, compared to an FDA-funded natural history dataset on DMD cardiomyopathy.
This positive FDA feedback is a critical step towards potential approval of deramiocel, offering a much-needed treatment option for DMD cardiomyopathy, a leading cause of death in DMD patients. Currently, treatment options are limited, and the progressive nature of this heart condition underscores the urgency for effective therapies. A successful approval would validate Capricor’s cell therapy approach and could pave the way for its application in other rare diseases.
The FDA’s recognition of the BLA’s completeness without significant deficiencies strengthens the likelihood of deramiocel’s approval. The upcoming advisory committee meeting will provide a platform to present the therapy’s efficacy and safety data, further contributing to the review process. Deramiocel, an allogeneic cardiosphere-derived cell therapy, has received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), along with RMAT and ATMP designations, respectively. Capricor has a commercialization agreement with Nippon Shinyaku for deramiocel in the United States and Japan, pending regulatory approval. Should the FDA grant approval, Capricor would also receive a Priority Review Voucher.
This progress positions deramiocel as a potential first-in-class therapy for DMD cardiomyopathy. The upcoming advisory committee meeting and the August PDUFA date are key inflection points that could significantly impact Capricor’s future, potentially transforming the treatment landscape for DMD patients facing life-threatening heart complications and solidifying Capricor’s position in the rare disease market.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
