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# Reviewers Still Negative on Sarepta Duchenne Drug
- URL: https://www.fdaweb.com/reviewers-still-negative-on-sarepta-duchenne-drug/
- Published: 2016-04-21T12:00:00.000Z
- Updated: 2026-09-14T20:56:29.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5135455

> FDA medical reviewers say that clinical data submitted by Sarepta Therapeutics to support its NDA for eteplirsen to treat Duchenne muscular dystrophy (DMD) “overall did not provide statistical evidence to support the efficacy” of the drug. The assessment is part of a [briefing document](http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM497063.pdf?ref=fdaweb.com) released in advance of a 4/25 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee to discuss the NDA.  
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> Sarepta is seeking accelerated approval for eteplirsen in patients with DMD who have mutations amenable to exon 51 skipping. The advisory committee meeting had been slated for 1/22 but was rescheduled due to a weather emergency. The briefing document notes that the materials released in advance of 1/22 contained a negative view of the drug and since then the company submitted additional information about clinical outcomes and its responses to what Sarepta said were “inaccuracies” in the FDA document. The latest briefing document incorporates the additional company information and rejects the characterization of the earlier document as containing inaccuracies.  
>  
> The reviewers said that the new information submitted by the company further increased concerns about the reliability of its clinical trial data.  
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> One issue deals with the three small studies the company submitted in support of its NDA. The agency reviewers wrote that while FDA is flexible in looking at treatments for diseases that have no current treatment options, “we cannot approve drugs for which substantial evidence of effectiveness has not been established.” The reviewers criticized Sarepta’s clinical study design and statistical analysis, arguing that the company over-estimated how much dystrophin was produced by the drug and how much role the drug played in meeting a clinical endpoint.  
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> There are reports that some 800 patients and members of their families will be at the advisory committee meeting and that CDER director **Janet Woodcock** will appear personally before the panel, indicating the pressure that is on the agency and the committee.  
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> CureDuchenne, a California nonprofit that provided early-stage funding for eteplirsen and supports its approval, reportedly released a statement expressing disappointment at the FDA reviewers’ position. “While we understand FDA needs to make sure drugs are safe and effective,” the group is quoted as saying, “we also know the consequence of inaction for those with Duchenne. We are hopeful for accelerated approval.”