Capricor Therapeutics (NASDAQ:CAPR) shares plummeted more than 26% after it reported the US Food and Drug Administration (FDA) has not approved its Biologics License Application for deramiocel, the company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
In a Complete Response Letter to Capricor, the FDA stated that the application did not meet the statutory requirement for substantial evidence of effectiveness and requested additional clinical data before reconsidering approval.
There were also outstanding items in the Chemistry, Manufacturing, and Controls section, though Capricor believes most have already been addressed.
Capricor CEO Linda Marbán expressed surprise at the FDA’s decision, noting that before the letter, the review had advanced without major issues.
The company plans to resubmit its application with new data from its ongoing Phase 3 HOPE-3 trial, which is expected to report topline results in the third quarter of 2025.
“We believe these data, if positive, along with our existing long-term clinical results showing cardiac stabilization, preservation of skeletal muscle function, and a consistent safety profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with DMD,” Marbán said.
“While this was an unexpected decision by the FDA, we remain committed to the DMD community to get deramiocel through the approval process.”
Shares of Capricor traded down 26.2% at about $8 late morning on Friday.