The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee was placed in an unusual position in its recent review of ataluren (Translarna, PTC Therapeutics), an agent for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene (nmDMD). The FDA had twice refused to accept the New Drug Application, but the company had filed it anyway.
Underscoring the contentiousness of the review, in opening remarks before the committee, Murad