Capricor Therapeutics received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, its lead cell therapy candidate for Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The FDA stated the BLA lacked substantial evidence of effectiveness and requires additional clinical data. The FDA also noted outstanding Chemistry, Manufacturing, and Controls (CMC) issues, though Capricor believes these were addressed in prior communications.
This CRL impacts the DMD patient community awaiting potential treatment options for this debilitating disease. Deramiocel held promise, particularly given its prior designations of Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT), and Rare Pediatric Disease. The delay in potential approval prolongs the unmet medical need for an effective therapy targeting DMD-associated cardiomyopathy, the leading cause of death in DMD patients. Further, it stalls Capricor’s potential receipt of a Priority Review Voucher, a valuable asset that can expedite future drug approvals.
The FDA has invited Capricor to a Type A meeting to discuss the path forward. Capricor plans to resubmit its BLA in Q3 2025, incorporating data from the ongoing Phase 3 HOPE-3 trial. This trial is a randomized, double-blind, placebo-controlled study involving 104 patients. The HOPE-3 data is crucial, as its positive results could address the FDA’s concerns about Deramiocel’s effectiveness.
This CRL represents a setback for Capricor. The company now faces the challenge of generating positive data from the HOPE-3 trial and successfully addressing the remaining CMC issues to secure eventual approval. The future of Deramiocel hinges on the strength of the upcoming clinical data and the subsequent interactions with the FDA. This situation underscores the complexities and inherent risks in drug development, particularly for rare diseases like DMD.
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.
