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U.S. lawmakers seek info on FDA's approval of Duchenne drug
March 15, 2017 / 8:10 PM / 9 months ago

U.S. lawmakers seek info on FDA's approval of Duchenne drug

March 15 (Reuters) - Two U.S. lawmakers on Wednesday sent a letter to the U.S. Food and Drug Administration, requesting information about the “unusual circumstances” surrounding the approval of Marathon Pharmaceuticals LLC’s drug for a muscle-wasting disorder.

Last month, Marathon paused the launch of its Duchenne muscular dystrophy drug after the lawmakers questioned the company why it priced the drug at $89,000 a year when patients had been able to import it for as little as $1,000.

"The high price was especially troubling in light of the incredibly lucrative benefits FDA has granted to Marathon," Senator Bernie Sanders and Representative Elijah Cummings wrote to acting FDA Commissioner Dr. Stephen Ostroff. (bit.ly/2mPlfqU)

Questions remain about the data the FDA used to evaluate the drug and why Marathon was given market exclusivity for a 20-year-old drug that the company did no significant research on, Cummings and Sanders said.

Independent Senator Sanders and Cummings, the top Democrat on the House of Representatives Committee on Oversight and Government Reform, had also questioned the exorbitant price tag.

Marathon’s drug, Emflaza, known generically as deflazacort, is a steroid, one of a class of drugs commonly used to treat Duchenne’s that patients could import for personal use because it was not available in the United States.

Last month, it won U.S. approval, in theory closing off that option.

The FDA will review the letter and respond directly to the lawmakers, a spokeswoman for the agency told Reuters. (Reporting by Ankur Banerjee in Bengaluru; Editing by Maju Samuel)

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