FDA requests more data on Capricor’s DMD cell therapy
Capricor completed its submission for FDA approval of deramiocel in DMD cardiomyopathy in January 2025. However, the FDA decided that the deramiocel submission fell short of the requirement for substantial evidence of effectiveness and they asked to see more clinical data. Capricor may be able to meet the request for additional clinical data quickly.
Capricor started a phase 3 trial in 2022 after meeting with the FDA to discuss the next steps. They opted against seeking approval on the strength of a phase 2 dataset that was smaller than planned. After three years of data from an open-label extension to the phase 2 trial showed sustained improvements in heart function, they made the submission which has just been rejected.
Capricor expects to have data from a double-blind, placebo-controlled phase 3 trial of 104 patients in the third quarter of 2025. Capricor CEO Linda Marbán, Ph.D., said in a statement that the company believes the phase 3 results, plus existing data, “could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD.”
