Yesterday, the Food and Drug Administration made history, approving a drug to treat Duchenne muscular dystrophy that works by targeting the genetic mutation at the root of the disease.
The decision was unique for reasons that were not just scientific. Janet Woodcock, the director of the FDA’s Center for Drug Evaluation and Research overruled the protests of her own staff to approve the drug. One of her division’s top officials appealed the ruling, and the agency’s chief scientist largely backed his conclusions, saying that “by any meaningful objective standard” the medicine is unlikely to improve patients.
