Capricor Therapeutics said the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee is scheduled to meet on July 29, 2026, to review the company’s biologics license application for deramiocel in Duchenne muscular dystrophy.
The company said its application remains on track for the FDA’s August 22, 2026 PDUFA target date. Deramiocel’s filing is supported by Phase 2 HOPE-2 data, long-term follow-up from HOPE-2-OLE, and Phase 3 HOPE-3 results.
In HOPE-3, the therapy met its primary endpoint, PUL v2.0, and also hit the key secondary cardiac endpoint, LVEF, along with all other type I error-controlled secondary endpoints, according to the release.
Capricor also highlighted the size of the disease opportunity, saying Duchenne muscular dystrophy affects about 15,000 people in the U.S. The company said deramiocel has been administered to more than 250 human subjects across multiple clinical trials and has been the subject of more than 250 peer-reviewed publications.
The company said deramiocel has received orphan drug designation from both the FDA and the European Medicines Agency, along with RMAT designation in the U.S., ATMP designation in Europe and rare pediatric disease designation from the FDA. As a result of these announcements, the company's shares have moved -13.03% on the market, and are now trading at a price of $26.44. If you want to know more, read the company's complete 8-K report here.
