STAT Plus: The FDA continues to struggle with the implications of approving Sarepta’s drugs
New documents released Wednesday lay bare the stark divide within the Food and Drug Administration regarding the approval of Vyondys 53, a medicine to treat boys with Duchenne muscular dystrophy made by Sarepta Therapeutics.
Vyondys was rejected in August. At the time, Sarepta said that the FDA had made its decision based on a risk of kidney toxicity seen in animal studies and the risk of infections. The new documents, though, reveal that the FDA’s concerns went deeper, to the lack of evidence available to back up the medicine’s theoretical benefit and to the difficulty of weighing a drug’s efficacy against its risks when both are difficult to measure.
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