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ARTICLE | Politics & Policy

Sanders, Cummings press FDA on Marathon's Emflaza

March 15, 2017 11:49 PM UTC

In a letter to FDA Acting Commissioner Stephen Ostroff, Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) asked the agency to defend its approval of Emflaza deflazacort from Marathon Pharmaceuticals LLC (Northbrook, Ill.) and the benefits the company has received via incentive programs. Marathon has faced criticism since pricing the steroid at $89,000 per year following its approval last month to treat Duchenne muscular dystrophy (DMD), although it had long been available via importation at a small fraction of the price.

The lawmakers questioned the clinical data used to support Emflaza's approval, asking if it is "standard practice for FDA to rely on 20-year-old efficacy data," and how many times its used such data in the past 15 years. They requested evidence showing the integrity of the "decades-old efficacy trial data," and asked if the agency took any measures to verify the data...

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