Capricor Therapeutics announced on Mar. 11 that the U.S. Food and Drug Administration will review its application for full approval of deramiocel, an investigational cell therapy for Duchenne muscular dystrophy, with a decision expected by Aug. 22.
The FDA's renewed review follows the agency lifting its previous complete response letter, which had rejected Capricor’s initial submission in July 2025 due to what it called a lack of substantial evidence of effectiveness. The new review could have significant implications for patients with Duchenne muscular dystrophy, a rare disease with limited treatment options.
Deramiocel is composed of cardiosphere-derived cells from the heart and is designed to help preserve cardiac and skeletal muscle function by modulating immune response and reducing fibrosis. Capricor tested the therapy in the Phase 2 HOPE-2 study, reporting meaningful improvements in upper limb function among patients. The company later resubmitted its drug package in December 2025, including data from the Phase 3 HOPE-3 trial that showed significant benefits in upper-limb and cardiac function compared to placebo, as well as slowed progression of skeletal muscle disease.
Capricor’s shares rose by 9% to $33.40 following the announcement. The company said it has not been notified by the FDA of any potential issues that could affect deramiocel’s review process.
If approved, Capricor may receive a priority review voucher under the FDA’s rare pediatric disease program. Analysts suggest that upcoming leadership changes at the FDA could also impact regulatory decisions affecting rare diseases like Duchenne muscular dystrophy.
The FDA has stated it remains committed to notifying companies about regulatory actions before sharing information publicly or with media.
