Sarepta's shares fall 40 percent as FDA request delays filing for Duchenne muscular dystrophy drug eteplirsen

Shares in Sarepta Therapeutics slipped as much as 40 percent Monday after the company announced that it plans to seek FDA approval of the Duchenne muscular dystrophy (DMD) drug eteplirsen by mid-2015, delayed from a previous timeline of the end of this year. The company noted that the regulator asked for further data to be included in the filing.

Specifically, Sarepta noted that the FDA requested results from an independent assessment of dystrophin images and 168-week clinical data from the mid-stage Study 202, for which the drugmaker reported 144-week results in July. The agency also asked the company to provide three-month data from at least 12 to 24 newly exposed patients, available results from other studies, even if they exposure time was less than three months, as well as patient-level natural history data. Sarepta added that some of the FDA's questions focus on the methods of measuring dystrophin.

The company also stated that the regulator stressed that further discussions are needed to clarify what would constitute a complete marketing application. "We are committed to satisfying the FDA's updated requests for these specific data to be included as part of an NDA submission and will continue to work with the agency toward the goal of a complete and acceptable NDA filing," commented Sarepta CEO Chris Garabedian.

Earlier this month, Prosensa started a rolling submission with the FDA seeking approval of its DMD therapy drisapersen.

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