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Sarepta scientists, FDA clash over efficacy of experimental drug
Boston Globe

Scientists from Sarepta Therapeutics Inc. and the Food and Drug Administration clashed Monday morning over their conflicting interpretations of findings from Sarepta’s clinical trial of an experimental drug to treat Duchenne muscular dystrophy.

Their differences, laid bare at an overflow meeting of an FDA advisory committee, puts the onus on the influential panel of outside medical experts to determine if the Cambridge biotech company’s drug candidate is effective enough for them to recommend approval in the United States.

The meeting is the most closely watched FDA hearing on a proposed therapy in years, with advocates arguing that failure to approved the drug could not only worsen the quality of life for Duchenne patients but also discourage biotechs from developing drugs to treat other rare diseases.

 

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