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SRPT: Sarepta Therapeutics sees setback in Eteplirsen drug application
8:59 AM ET 10/27/14
By Erin McCarthy

Sarepta Therapeutics Inc. said the U.S. Food and Drug Administration is requiring additional data for the new drug application for its Duchenne muscular dystrophy treatment eteplirsen.

As a result, Sarepta (SRPT) plans to submit a new drug application for the treatment by midyear 2015, later than the company had previously planned. Earlier this year, the company said it had expected to submit the new drug application by the end of this year.

Shares of the company dived more than 33% in premarket trading in response.

The company and the FDA held a type B, pre-investigational new-drug application meeting last month. In the meeting minutes, the FDA said that the required additional data includes the results from an independent assessment of dystrophin images and the 168-week clinical data from study 202, according to Sarepta.

The FDA's guidance also requests more specific data, including a minimum duration of safety in new patients exposed to eteplirsen, patient-level natural history data to be obtained by Sarepta from independent academic institutions, and MRI data from a recent study conducted by an independent academic group.