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Sarepta's Additional Data Doesn't Sway FDA On Eteplirsen

This article was originally published in The Pink Sheet Daily

Executive Summary

Agency reviewers raise more concerns about efficacy of proposed Duchenne muscular dystrophy treatment despite new information.

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EMA and payers want to know more about daily life improvements from US FDA-approved Duchenne muscular dystrophy treatment.

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Witnesses call for political pressure on FDA to approve the Duchenne drug; patients and advocates also urge general approval reciprocity with Europe and incentives for pediatric trials; agency was not invited to testify.

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