FDA Panel Rejects Duchenne Drug in 9-3 Vote Over Data Integrity Dispute

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 against recommending approval of Capricor Therapeutics' deramiocel for Duchenne-related cardiomyopathy, concluding the company failed to provide "substantial evidence" of effectiveness. The contentious meeting centered on Capricor's Phase III HOPE-3 trial, which the company claimed showed the therapy slowed upper limb decline in 106 boys and young men aged 10 to 22 with advanced Duchenne muscular...