Capricor Therapeutics said the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee is set to meet on July 29, 2026, to review the company’s biologics license application for deramiocel in Duchenne muscular dystrophy.
The company said its PDUFA target action date remains Aug. 22, 2026, leaving 24 days between the advisory committee meeting and the FDA decision deadline.
Capricor said the application is backed by its phase 2 HOPE-2 trial, long-term data from HOPE-2-OLE, and phase 3 HOPE-3 results. In HOPE-3, the therapy met its primary endpoint, PUL v2.0, its key secondary cardiac endpoint, LVEF, and all other type I error-controlled secondary endpoints.
The company said deramiocel has been studied in more than 250 peer-reviewed publications and administered to over 250 human subjects across multiple clinical trials.
Capricor also said deramiocel has received orphan drug designation from both the FDA and the European Medicines Agency, RMAT designation in the U.S., ATMP designation in Europe, and rare pediatric disease designation from the FDA. Following these announcements, the company's shares moved -13.03%, and are now trading at a price of $26.44. Check out the company's full 8-K submission here.
