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# FDA Extends Review on Sarepta Duchenne Drug
- URL: https://www.fdaweb.com/fda-extends-review-on-sarepta-duchenne-drug/
- Published: 2016-02-08T12:00:00.000Z
- Updated: 2026-09-15T02:33:58.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5134700

> FDA has extended by three months its review of a Sarepta Therapeutics NDA for eteplirsen, indicated for treating Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The new user fee review target date is 5/26\. The agency recently notified Sarepta that a 1/8 submission of four-year clinical effectiveness data, which included additional six minute walk test and loss of ambulation data compared to a historical control, has been designated as a major amendment to the NDA. A rescheduled date for an FDA Peripheral and Central Nervous System Advisory Committee meeting on the drug that was canceled due to snow in January has not yet been determined, the company says.  
>  
> FDA has been under pressure by patient advocates and Congress after it reversed course in 2013 and told Sarepta that an NDA for eteplirsen would be premature for treating DMD ([see story](http://fdaweb.com/login.php?sa=v&aid=D5126887&searchWords=Sarepta&cate=S&stid=%241%24N25.SL5.%246Mm7PAHxXg4QxrweHvxgB0&ref=fdaweb.com)). At the time, the agency cited recent developments that caused some alarm, including a failed study with a competitive product and recent natural history data in DMD, according to the company. The agency said the new data raise “considerable doubt” about “both the dystrophin biomarker and the supportive clinical efficacy assessed on the six-minute walk test (6MWT) in the Phase 2b clinical study of eteplirsen,” according to the company.  
>  
> In 2014, CDER director **Janet Woodcock** said the agency was willing to explore the use of all potential pathways for approving drugs for DMD, including accelerated approval, as appropriate. That is the [response](https://petitions.whitehouse.gov/response/drug-approval-pathways-and-duchenne-muscular-dystrophy?utm%5Fsource=wethepeople&utm%5Fmedium=email&utm%5Fcontent=duchenne-response) she gave to a petition on the White House’s Web site that has garnered over 100,000 signatures in support of the agency’s accelerated approval pathway for such products.   
>  
> Woodcock’s response said the agency’s “ongoing analyses of eteplirsen and other drugs for the treatment of Duchenne muscular dystrophy are based on rigorous assessments by a large multi-disciplinary team of scientists. Although our assessments are rigorous and extensive, we recognize the urgency of the needs of patients with Duchenne muscular dystrophy, and we carry out our analyses expeditiously.”