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# FDA Asks for More Data on Duchenne Drug NDA
- URL: https://www.fdaweb.com/fda-asks-for-more-data-on-duchenne-drug-nda/
- Published: 2016-06-07T12:00:00.000Z
- Updated: 2026-09-14T21:08:10.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5135856

Sarepta Therapeutics and its eteplirsen for treating Duchenne muscular dystrophy received a shot in the arm 6/6 when, in an apparent compromise with FDA, the company agreed to an agency request that it provide dystrophin data, as measured by western blot, from biopsies already obtained from an ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing NDA evaluation. The company plans to submit data from13 patient biopsy samples, at baseline and week 48, to the agency over the next few weeks. In a [statement](http://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=2175522&ref=fdaweb.com), Sarepta said it expects this will “facilitate a prompt decision on the NDA by the agency.” Eteplirsen is designed to address the underlying cause of Duchenne’s by restoring the dystrophin messenger RNA (mRNA) reading frame, and enabling the production of a shorter, functional form of the dystrophin protein, according to the company.

Last month, FDA extended its NDA review, and the submission has become a politically charged issue, with U.S. lawmakers weighing in and urging the agency to exercise its flexibilities when making a review decision. At the time, the agency reportedly communicated thatit will continue to work past the 5/25 user fee goal date and strive to completeits work in as timely a manner as possible.

In April, FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 7 to 6 ([see story](https://www.fdaweb.com/fda-panel-split-vote-not-encouraging-for-duchenne-drug/)) that a Sarepta Therapeutics NDA did not provide substantial evidence from adequate and well controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit in Duchenne patients. The vote was seen as a setback for the company’s bid to gain accelerated approval for the drug. A separate 7 to 3 vote by the panel recommended that data were not sufficient to meet traditional approval standards.However, before those votes, CDER director **Janet Woodcock** obliquely [hinted at a more positive assessment](http://www.barrons.com/articles/slim-sarepta-hopes-rest-with-top-fda-official-1461695932?ref=fdaweb.com) of the drug in an unusual appearance she made before the panel.