Muscular dystrophy drug cost escalates to $89000 per year

14 February, 2017, 00:37 | Author: Lorraine Moran

"We hope that this treatment option will benefit many patients with DMD".

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.

There's a new Duchenne muscular dystrophy (DMD) drug on the scene-at least, technically speaking. It can be administered to patients 5-years-old and above. As the condition progresses, chances of life-threatening conditions developing involving the respiratory system and the heart grow. "This also seems to be another example of gaming the Orphan Drug Act, which was meant to try and encourage research into new therapeutic entities for people who have rare diseases - and it doesn't seem like this is that". DMD occurs in about one of every 3,600 male infants worldwide. In the U.S.it affects about 15,000 people. Parent Project Muscular Dystrophy has received money from Marathon to support its conferences. Marathon said that as of now just around seven to nine percent of those afflicted with Duchenne have access to deflazacort. Its content is produced independently of USA TODAY.

Emflaza is marketed by Marathon Pharmaceuticals.

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According to The Wall Street Journal, the FDA-approved deflazacort would be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the US.

The drug wasn't previously sold in the USA because there was no company that would think it was profitable enough to warrant the effort of getting FDA approval. Prednisone, another steroid, is frequently prescribed for the disease though it does not have official FDA approval. This voucher can be used to get a drug reviewed faster; it can also be sold to other company for hundreds of millions of dollars. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began.

Deflazacort is the usual treatment for DMD across the world, but it is not a cure. Twelve weeks into the study, patients, who were taking deflazacort, showed marked improvements in muscle strength compared to those were taking a placebo. In general, the muscle strength was maintained among those taking the drug until the end of the 52-week study. 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.

The most common adverse effects with Emflaza included Cushingoid appearance, weight gain, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, and central obesity.

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