An FDA panel voted 9-3 against it. Does the FDA approve Capricor's Duchenne therapy anyway by Aug 22?
78% of users predicted NO — the community got this one right. 23 predictions cast.
The FDA did not approve Capricor Therapeutics' cell therapy deramiocel for Duchenne muscular dystrophy-associated cardiomyopathy by its original Aug. 22, 2026, PDUFA target date. Instead, on Aug. 24 the company announced the agency extended its review by three months, pushing the target action date to Nov. 22, after Capricor submitted a major amendment to its Biologics License Application.
The amendment included 24-month open-label extension data from the Phase 3 HOPE-3 trial and additional analyses supporting a narrower, refined indication focused on preserving upper-limb function, rather than the broader cardiomyopathy indication an FDA advisory committee had reviewed. That committee voted 9-3 in July that existing evidence did not provide substantial evidence of effectiveness, though the panel's recommendation was nonbinding. HOPE-3 had shown deramiocel slowed decline on a standard upper-limb function measure by 54% and slowed decline in heart pumping capacity by 91% versus placebo.
The extension keeps deramiocel's approval prospects alive but delays any decision into the fall.
Predict Six's community had assigned only a 22% chance of approval by Aug. 22, correctly anticipating that the FDA would not approve the therapy by that date.