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Limbo Land For Sarepta, Duchenne And Rare Diseases

Executive Summary

While investors were hopeful the FDA's delay in issuing its verdict for Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy medicine eteplirsen may mean the agency is not prepared to reject it, the postponement may create more doubt and uncertainty about the path forward in rare disease drug development in general.

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Data Trumps Anecdotes, Emotion At Sarepta Panel

At a highly charged April 25 meeting of the FDA's Peripheral and Central Nervous System (PCNS) Advisory Committee, which concluded with angry shouting from parents and patient advocates, a majority of panelists said Sarepta Therapeutics Inc. failed to provide sufficient efficacy data for its Duchenne muscular dystrophy drug eteplirsen to win US approval – accelerated or standard, although the votes were much closer on the former than on the latter question.

Sarepta Lashed Again; More Duchenne Market Doubt

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Duchenne Market Gets Bleaker With Sarepta Eteplirsen Delay

Adding to the already bleak outlook for the Duchenne muscular dystrophy (DMD) market, Sarepta Therapeutics Inc. on Feb. 8 disclosed the FDA was delaying its verdict on the company's experimental drug eteplirsen until May 26 – citing a "major amendment" to the new drug application (NDA) submitted by the company.

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