Health Care

FDA Approves Duchenne Muscular Dystrophy Drug

FDA Approves Duchenne Muscular Dystrophy Drug

Marathon Pharmaceuticals Chief Financial Officer Babar Ghias said, "For patients, the cost of the drug - which goes by the brand name Emflaza, would be "zero to low out-of-pocket expense", because of insurance and financial assistance programs that the company will offer".

Before the approval, patients had to get emflaza from overseas for $1,200 a year. The disorder is caused when there is an absence of the protein dystrophin, which helps to keep the muscles intact, according to the FDA. Marathon Pharmaceuticals will sell the FDA-approved deflazacort drug under the brand name Emflaza for almost $89,000 for a year's worth of treatment. DMD commonly manifests in individuals without known family histories of the condition and mainly affects boys. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States. Parent Project Muscular Dystrophy has received money from Marathon to support its conferences.

Corticosteroids are a common treatment option for DMD around the world but this is the first time that the FDA has approved any drug in the class to treat the condition and also the first time that Emflaza was approved for any kind of use. But until now it hasn't been widely available in the USA because it wasn't approved by the FDA. Life expectancy and disease severity vary but many patients usually succumb to DMD in their 20s or 30s.

Trump's wall to cost over $21bn, says gov't report
The third phase would cover an unspecified 1,080 miles (1,728 km), essentially sealing off the entire U.S. -Mexico border. Trump has said Congress should fund the wall upfront, but that Mexico will reimburse US taxpayers.

"Now the hope is at least all children will have access to an actual FDA-approved therapy for Duchenne", said Craig McDonalds, a professor at the University of California.

The drug wasn't previously sold in the US because there was no company that would think it was profitable enough to warrant the effort of getting FDA approval.

The approval of the drug, which will carry a list price of $89,000 for a year's supply, comes as pharmaceutical companies face growing pressure to hold down medication prices. However, U.S. patients have been importing the drug from foreign countries since the 1990s, after clinical studies demonstrated its potential for reducing inflammation with fewer adverse effects than another steroid. It is a practice that has prompted congressional investigations for other companies in the past, most notably, Turing Pharmaceuticals, the firm formerly run by one time hedge-fund manager Martin Shkreli, who took a drug used to treat AIDS patients, and increased 5,000 percent from $13.50 a pill to $750. In addition, although not statistically controlled for multiple comparisons, patients on deflazacort appeared to lose the ability to walk later than those treated with placebo.


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