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FDA Duchenne Drug Review Smacks Sarepta

This article was originally published in Scrip

Executive Summary

FDA drug reviewers raised considerable doubts about whether Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy (DMD) drug eteplirsen, which it wants to market as Exondys under an accelerated approval, was effective in treating the disease – calling into question whether the investigational medicine actually increased the levels of dystrophin, a protein essential for normal muscular structure and function and the lack of which is at the heart of the condition.