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# Senate Committee Urges FDA Approval of Duchenne Drug
- URL: https://www.fdaweb.com/senate-committee-urges-fda-approval-of-duchenne-drug/
- Published: 2016-05-23T12:00:00.000Z
- Updated: 2026-09-14T21:04:43.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5135740

The Senate Committee on Homeland Security and Governmental Affairs has written FDA commissioner **Robert Califf** urging the agency to exercise its flexibilities when making a review decision this week on a Sarepta Therapeutics NDA for eteplirsen and its use in treating Duchenne muscular dystrophy. In April, the agency’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. The 5/20 [letter](http://www.hsgac.senate.gov/download/senators-johnson-and-coats-letter-to-fda-commissioner-califf?ref=fdaweb.com) to Califf encourages FDA to “prioritize the patient perspective” when making a decision on the submission. It also complains that the questions given to advisory committee member were posed in such a way to make it difficult for them to vote favorably on the NDA. “Patients are crying out for the FDA to hear them: they are engaged and knowledgeable and only want the agency to do what is already within their power,” it says. “We fully support their perspective.”