In September 2016, the FDA approved eteplirsen, a new treatment for Duchenne muscular dystrophy, a previously-untreatable fatal childhood disease. Eteplirsen received accelerated approval, which is available for drugs treating “serious or life-threatening diseases” that provide “meaningful” benefit over existing therapies. Sarepta satisfied FDA’s standard using a twelve-patient clinical trial and the surrogate endpoint of dystrophin production.

The primary scientific bodies tasked with overseeing the review process concluded that eteplirsen should not be approved. Dr. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, overrode reviewers’ objections and granted eteplirsen accelerated approval. However, Dr. Ellis Unger disagreed with Woodcock’s conclusion. Unger’s appeal sent the issue to Dr. Robert Califf, the FDA Commissioner, who deferred to Woodcock. Almost as soon as eteplirsen was approved, multiple FDA officials—including Califf—called for one of the two studies to be retracted because it was misleading.

Questions about the drug’s effectiveness, the FDA “civil war” behind the scenes, and concerns about the design of the clinical trials led to extensive media coverage. Working with Charles Seife, a journalist and NYU professor, we submitted a FOIA request to FDA seeking certain correspondence, documents, and clinical data that could shed light on the unorthodox approval process. The FOIA requested “expedited processing”—which requires a determination to be made in 10 days and production to be made “as soon as praciticable”—on the basis that there was an urgent need to inform the public about the issues surrounding the drug’s approval.

Charles Seife and the FDA were able, under judicial direction, to negotiate a schedule for the production of responsive documents and a grant of expedited processing from the FDA, although it was on the basis of the agency’s discretion rather than based on the urgency of the request. Production began on July 24, 2017, with the “Jenkins Memo,” the contents of which Seife chronicled in an article for Undark.[1] A second production included communications regarding the attempted retraction of an article in Annals of Neurology by the FDA review team which Seife chronicled in an article for Retraction Watch.[2]

Production was complete in December 2017. The case is at the summary judgment phase, with our motion due on May 21, 2018.

[1] https://undark.org/article/fda-eteplirsen-janet-woodcock/
[2] http://retractionwatch.com/2017/08/21/released-fda-docs-reveal-details-a...

 

Key Documents Include:

Complaint (filed May 25, 2017)
Seife’s Brief in Support of Cross Motion for Summary Judgement (filed May 29, 2018)
Seife’s Combined Reply Memorandum of Law (filed August 30, 2018)