Duchenne Muscular Dystrophy: Second Product Isn't The Charm

FDA reviewers say patients treated with Sarepta's eteplirsen seemed to follow similar disease course as Duchenne muscular dystrophy natural history, casting doubts on product's approvability.

Like its competitor drisapersen, Sarepta Therapeutics Inc.'s eteplirsen will not enter its advisory committee meeting brimming with optimism for approval after FDA reviewers identified significant efficacy problems.

Eteplirsen, seeking an indication for Duchenne muscular dystrophy patients who have a confirmed dystrophin gene mutation amenable to exon 51 skipping, will go before the Peripheral

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