New docs spotlight the FDA’s failed attempt to get Sarepta’s flawed Duchenne study retracted

Robert Califf

One of the reasons why Sarepta $SRPT had such a hard time getting its Duchenne drug through the FDA rested on a published study that regulators had concluded was based on unreliable data that led to erroneous conclusions.

That 48-week study of Sarepta’s would prove to be a major sticking point for the FDA, spurring team leader Ellis Unger and FDA commissioner Robert Califf to outline their argument to the editor of the Annals of Neurology, Clifford Saper.

According to an update from Retraction Watch, which looked over some of the documents unveiled by a FOIA lawsuit against the FDA, Saper ultimately decided to let the FDA execs write a letter or accept an “Erratum” from the paper’s authors, who described it as a comparison of the two analyses.

Califf and Unger chose the letter, and as Retraction Watch notes, the heavily criticized paper stands as published — unstained by the FDA’s opinions or the dispute over its accuracy.

Saper told Retraction Watch:

The point of this entire thing was to get the record straight….It is now in the literature.  There is no point in publishing the same thing again. Or in discussing the same thing again, and again, and again, and again….

Janet Woodcock

The controversy over eteplirsen, though, continues to bubble as new documents have been published from the lawsuit highlighting heated top-level discussions about the role Janet Woodcock played in getting the drug approved.

Woodcock not only appeared biased to some, she also nixed any objections in pushing through an approval. Now the drug is sold for hundreds of thousands of dollars a year, to Duchenne families who swear by the drug, even though there is no hard data to support its efficacy.

And don’t expect this to be the final word, either. This is one argument that won’t play out until actual data is available. And that will take years.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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