Capricor Therapeutics says the United States Food and Drug Administration (FDA) is willing to review an amendment containing 24-month HOPE-3 data for deramiocel, keeping the experimental Duchenne muscular dystrophy cell therapy under active consideration despite a strongly negative advisory committee vote. The company intends to narrow the regulatory case from Duchenne-associated cardiomyopathy to preservation of
Capricor Therapeutics faces an August 22 FDA decision for deramiocel after advisers voted that the available evidence did not establish effectiveness in Duchenne muscular dystrophy cardiomyopathy. The disputed case now turns on statistical methods, the treatment indication and whether supportive upper-limb results can overcome unresolved cardiac evidence.
FDA advisers voted 9 to 3 against deramiocel’s Duchenne cardiomyopathy evidence after challenging the Phase 3 analysis and trial population.
Capricor Therapeutics has secured a July 29 FDA advisory committee meeting for deramiocel after a positive Phase 3 trial revived the previously rejected Duchenne muscular dystrophy application. The therapy now has controlled evidence of skeletal and cardiac benefit, but regulators must still weigh endpoint interpretation, long-term external comparisons, manufacturing reliability and whether the proposed label