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Marathon to charge $89000 for DMD drug after 70-fold increase

Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) sent a letter to Marathon CEO Jeffrey Aronin on Monday, accusing the company of abusing a rule meant to incentivize treatments for diseases which have none. Recently, Marathon acquired the rights to historical clinical trial data from the 1990s and completed some additional analyses to gain approval from the Food and Drug Administration to sell the medication in the United States. In an interview last week, the company's chief financial officer, Babar Ghias, said Marathon took the pricing issue "very seriously" and that its goal was to ensure patients can access the drug for zero to low cost.

The practice has prompted congressional investigations and hearings into companies including Valeant Pharmaceuticals International Inc. and Turing Pharmaceuticals LLC, the firm formerly run by onetime hedge-fund manager Martin Shkreli.

Here is the statement from the FDA.

Tracy also noted that while deflazacort is useful in DMD - as are other steroids, including prednisone, which is generic and has some efficacy evidence behind it - it is a purely symptomatic treatment, providing improved muscle strength.

Last week, Marathon earned approval for deflazacort, a steroid that has always been available outside the USA, to treat a devastating, fatal muscle deteriorating disease called Duchenne muscular dystrophy.

"This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy, " Billy Dunn, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research, said in the press release. Parent Project Muscular Dystrophy has received money from Marathon to support its conferences.

DMD is the most well-known kind of muscular dystrophy that caused by an absence of dystrophin, a protein that keeps muscle cells in place. DMD affects about one of every 3,600 male infants globally, according to the FDA.

People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease continues to progress, patients can develop life threatening respiratory and heart related conditions. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

The FDA approved Emflaza for all DMD patients five years and older. According to scientists, at week 12 the patients who were under deflazacort showed improvements in their muscle strength compared to those who were taking a placebo.

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