Capricor Therapeutics (NASDAQ: CAPR) announced that the U.S. Food and Drug Administration has lifted a previously issued Complete Response Letter and resumed review of its Biologics License Application for Deramiocel, a cell therapy for treating Duchenne muscular dystrophy cardiomyopathy. The FDA has set a new PDUFA target action date of August 22, 2026.

The submission has been classified as a Class 2 resubmission. Capricor received a Complete Response Letter from the FDA in July 2025. Following the company's submission of data and supporting documentation from the HOPE-3 clinical trial, the FDA resumed its review and assigned the new target date.

The FDA has not identified any potential review issues in its response to the company at this time, according to the press release statement.

Deramiocel consists of allogeneic cardiosphere-derived cells that target cardiac and skeletal muscle function in muscular dystrophies. The therapy has received Orphan Drug Designation from both the FDA and European Medicines Agency, along with Regenerative Medicine Advanced Therapy designation in the U.S.

Capricor expects to be eligible for a Priority Review Voucher upon potential approval of Deramiocel. The company has entered into an agreement with Nippon Shinyaku Co., Ltd. for exclusive commercialization and distribution of Deramiocel in the United States and Japan, subject to regulatory approval.

Duchenne muscular dystrophy affects approximately 15,000 individuals in the United States and primarily impacts boys. The genetic disorder causes progressive muscle degeneration affecting skeletal, respiratory, and cardiac muscles, with cardiomyopathy and heart failure being the leading cause of death.