Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy

The authors critique the FDA’s approval of eteplirsen for Duchenne muscular dystrophy, noting that the pivotal trial involved only 12 patients, failed to show clinically meaningful dystrophin production, and relied on post hoc analyses that excluded deteriorating patients to generate favorable results, while the FDA overruled both its scientific staff and advisory committee.