Testimony before the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Regarding SRP-9001 (delandistrogene moxeparvovec) for the Treatment of Duchenne Muscular Dystrophy
In testimony before the Food and Drug Administration’s (FDA’s) Cellular, Tissue and Gene Therapies Advisory Committee, Public Citizen urged the committee to reject accelerated approval for SRP-9001 (a gene therapy) for Duchenne muscular dystrophy because the sponsor-presented data did not support the drug’s effectiveness and because of concerning regulatory history for related drugs.