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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  • we all understand Exondys is the precedent that is driving Biogen’s filing of their Alzheimer’s drug, right? Super-high unmet need, lots of advocacy screaming for treatment. Without that precedent, no way Biogen would be trying to jam this through. And their strategy will probably work. FDA has set a terrible precedent.

    • The price is just south of one million dollars, based on body weight. The drastic changes from a prove-it system to a trail-and-error system will maximize revenues as long as companies can drag out the trials and bankrupt vulnerable families using a fake drug. How horrible that corporations have replaced humans in terms of degree of federal protection.

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