Northbrook company's muscular dystrophy drug wins FDA approval

A drug company may be facing some heat after doing something similar to Martin Shkreli, the hated man accused of jacking up the price of a life-saving drug just to make some more money.

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. That drug, however, only helps about 13 percent of Duchenne patients, those with a specific genetic mutation, said Tim Cunniff, Marathon executive vice president for research and development.

According to FDA, Emflaza works by reducing inflammation and lessening the movement of the immune system, and it is usually used to treat DMD over the world.

Ghias also says that that after rebates and discounts, the price will drop to $54,000, and with insurance and financial programs most patients will pay "zero to low out-of-pocket expense, " according to the Washington Post.

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Duchenne's, a devastating muscle-wasting disease, affects some 15,000 patients in the United States, mostly young boys. The top 100 orphan drugs in the USA cost an average of $111,820 a year per patient in 2014, according to a report by Evaluate, a market research firm.

He said Marathon is pricing the drug at $89,000 because of its usefulness for a small population, because of the six years Marathon worked to bring the drug to market and to perform further research on it. Marathon is a 6-year-old company that focuses on rare neurological diseases.

A physical therapist treats a patient with DMD.

"We urge you to significantly lower your price for this drug before it goes on the market [in March]", Sanders and Cummings wrote.

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In addition to winning these incentives for bringing the older treatment to the United States market as an orphan drug, Marathon also won a lucrative priority review voucher, which have sold for as much as $350 million.

The Food and Drug Administration approved the drug for use last week, after Marathon received a rare pediatric disease priority review voucher. By week 52, patients in the Emflaza group maintained an overall stability in average muscle strength. It improves muscle strength.

The FDA also granted Emflaza priority review and fast track and orphan drug designations. Patients lack a protein called dystrophin that's essential for healthy muscle, and eventually lose the ability to walk, stand and breathe.

Other side effects that are less common include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones and vision problems such as cataracts.

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(Copyright © 2015. All Rights Reserved.)
 
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