US FDA reveals discussions with Sarepta over eteplirsen

In what is viewed as an unusual step, the US Food and Drug Administration made a website posting of its discussions with biotech firm Sarepta Therapeutics (Nasdaq: SRPT) on its investigational Duchenne muscular dystrophy (DMD) drug.

The FDA noted that, on October 27, 2014, Sarepta Therapeutics released a statement and had a conference call regarding guidance received from FDA in a September 2014 meeting regarding its planned New Drug Application for eteplirsen, to treat patients with DMD (The Pharma Letter October 28). To the extent allowed by laws restricting release of confidential information about experimental drugs, the FDA says it is addressing questions the agency has received from DMD patients, their families, and others in the community who are concerned about the timing of the filing of an NDA for eteplirsen.

Over the past several years, the FDA has worked extensively with Sarepta on the development of eteplirsen, and provided guidance with respect to the data that would be necessary to determine whether it is effective and support filing of an NDA. Following a meeting with FDA last April, Sarepta announced on April 21, 2014, that “with additional data to support the efficacy and safety of eteplirsen for the treatment of DMD, an NDA should be fileable.” Sarepta also announced at that time that the FDA had communicated that there were areas of concern in the existing database, and that FDA had provided Sarepta with “examples of additional data and analyses that, if positive, would be important to enhance the acceptability of an NDA filing….” Sarepta announced at the time its plans to submit an NDA for eteplirsen by the end of 2014.