The FDA rejected Capricor Therapeutics' application for deramiocel, a cell therapy for cardiomyopathy associated with Duchenne muscular dystrophy (DMD), citing lack of substantial evidence of effectiveness and the need for additional clinical data13.
Capricor disputed the FDA's interpretation of their efficacy data, specifically the HOPE-2 trial, arguing that the agency misinterpreted the results and the trial actually met its primary efficacy endpoint when analyzed with what Capricor asserts were appropriate statistical techniques5.
The HOPE-2 trial's endpoint was a change in neuromuscular function. Capricor claims that while a standard t-test did not show significance due to unmet statistical assumptions, a non-parametric analysis yielded a statistically significant result (p-value of 0.014)5.
The FDA's complete response letter cited both efficacy concerns and outstanding issues in Chemistry, Manufacturing, and Controls (CMC), although Capricor stated that most CMC issues had already been addressed125.
Capricor plans to resubmit deramiocel for FDA review with additional data from its ongoing Phase 3 trial, aiming to resolve the agency's efficacy concerns12.
The FDA and Capricor have scheduled a meeting to discuss a path forward, but the review process will reset and require several more months after resubmission1.
Sources:
1. https://www.biopharmadive.com/news/fda-reject-capricor-deramiocel-duchenne-cardiomyopathy/752773/
2. https://www.neurologylive.com/view/fda-hands-capricor-complete-response-letter-deramiocel-dmd-cardiomyopathy-treatment
3. https://www.statnews.com/2025/07/11/fda-rejects-capricors-cell-therapy-for-duchenne-muscular-dystrophy/
5. https://www.fiercebiotech.com/biotech/capricor-shares-response-cell-therapy-denial-after-fda-publicly-releases-rejection-letter