Rare Disease Report

FDA Responds to the Duchenne Community about Sarepta's NDA Submission

NOVEMBER 01, 2014
RDR Staff

Earlier this week, Sarepta’s hopes for a quick approval of eteplirsen to treat patients a subset of patients with Duchenne muscular dystrophy - and their stock - declined significantly when they announced the FDA had suggested to the company that additional data would be needed before the FDA would likely accept a new drug application (NDA). 

At that time, Sarepta stated they still plan to submit their NDA mid-year 2015 and if eteplirsen is given accelerated approval, they hope to get the drug approved as early as 2016. Sarepta had previously hoped to submit and NDA in late 2014.

All of that occurred on Monday.

For the rest of the week, it seems that the Duchenne community reached out to the FDA with a plethora of questions. In response, the FDA has issued a statement online  to help answer some of the Duchenne families’ concerns.

In the statement, the FDA wrote:

“FDA understands the considerable disappointment in the Duchenne community following Sarepta’s October 27 announcement that the previous time frame for submitting the NDA for eteplirsen cannot be met.”

However, the FDA was fairly adamant in clarifying that they have consistently been advising Sarepta since April 2014 that they would have to  provide more data and conduct more studies.

One concern that the FDA addressed involved rumors that the fraud was suspected.  The FDA wrote:

“As described by Sarepta in its October 27 statement, the need for additional data and analyses to support the NDA was reinforced by an FDA inspection of the clinical site where dystrophin analyses had been conducted.  It is important to note that the agency did not find any evidence of fraud at this site, as has been perceived by some.  FDA is concerned that the methods used to measure dystrophin were not adequately robust to support an NDA submission.  Thus, FDA provided Sarepta with detailed recommendations on how to improve these dystrophin analyses, and FDA’s most recent advice was consistent with the advice provided after the April 2014 meeting."

Another issue the FDA wanted to clarify concerned the possibility of a rolling NDA submission.  The FDA wrote:

“ FDA has expressed willingness to conduct a “rolling review” of Sarepta’s NDA.  Under a rolling review, companies can submit, and FDA can review, portions of an application as they are completed.  Once submission of all components is complete, the review clock begins.  FDA expects the NDA for eteplirsen will qualify for a priority review.”

Regarding the possibility that eteplirsen will get given accelerated approval, the FDA is willing to consider it following a public review. The FDA wrote:

 “FDA also plans to present the NDA for eteplirsen to a public advisory committee meeting before making a decision on approval.  This will afford FDA the ability to gain advice from outside experts and interested stakeholders on the adequacy of the data to support approval, including the possibility of “accelerated approval” – a mechanism to approve drugs in particular situations prior to the availability of definitive evidence of effectiveness.”

Finally, the FDA, like Sarepta, noted that they:

“understand the dire urgency of the situation and the importance of our actions to the DMD community.  FDA will continue to work with Sarepta in their efforts to provide the data it considers critical to FDA’s ability to review the NDA and reach a decision on approvability.”

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