FDA Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy

On July 11, 2025, the FDA rejected Capricor Therapeutics' marketing application for deramiocel, a cell therapy targeting cardiomyopathy associated with Duchenne muscular dystrophy (DMD)12345.

The FDA issued a Complete Response Letter, stating the application failed to provide substantial evidence of effectiveness and requested additional clinical data before approval can be considered12345.

Capricor's rejection came after the company received Priority Review and completed pre-licensure inspections without major issues2.

Deramiocel would have been the first approved therapy specifically for DMD-related cardiomyopathy and could have been used alongside other DMD treatments13.

The FDA also referenced some unresolved manufacturing issues, though Capricor claims most have been addressed3.

Capricor plans to provide new data from its ongoing placebo-controlled Phase 3 HOPE-3 trial involving 104 patients, with topline results expected in Q3 2025234.

The FDA offered a meeting to discuss next steps and indicated the review process would restart following a resubmission of the application, taking an additional six to ten months3.

Following the rejection, Capricor's share price dropped significantly4.

Sources:

1. https://www.statnews.com/2025/07/11/fda-rejects-capricors-cell-therapy-for-duchenne-muscular-dystrophy/

2. https://www.stocktitan.net/news/CAPR/capricor-therapeutics-provides-regulatory-update-on-deramiocel-bla-v4uaabb8plj3.html

3. https://www.biopharmadive.com/news/fda-reject-capricor-deramiocel-duchenne-cardiomyopathy/752773/

4. https://www.fiercebiotech.com/biotech/fda-rejects-capricors-dmd-cell-therapy-asks-more-data

5. https://www.biospace.com/fda/capricor-plunges-on-fda-rejection-of-dmd-cell-therapy