FDA approves costly drug to fight Duchenne Muscular Dystrophy

FDA approves costly drug to fight Duchenne Muscular Dystrophy

The company intends to charge $89,000 a year for a drug which has been available in Canada, the United Kingdom and Europe for decades for $1,000-$1,600 a year, according to the Wall Street Journal.

It commonly implies the medication will be secure by insurance, and the evidence that is utilized to support the approval may induce doctors to use the medications. "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". Image credit: USA Today.$89,000 a year to treat the disease in the U.S.

Marathon anticipates a smooth transition for patients ready getting deflazacort from overseas pharmacies. Before now, this drug-known as deflazacort-has been available to patients who suffer from a quite devastating and generally fatal disease, Duchenne muscular dystrophy. However, the drug was never approved by the FDA as no companies thought it profitable enough to jump through the regulatory hoops necessary to sell it in the U.S.

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Marathon Pharmaceutical's deflazacort, a steroid, aims to improve the lives of patients with Duchenne muscular dystrophy by improving muscle function, potentially allowing them to walk, feed themselves and control their wheelchairs longer. 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.

However, the price hike underscores a problem with American pharmaceutical companies exploiting the orphan drug label to reap massive profits from medicine. This is the 9th time that the FDA grants the pediatric disease priority voucher. Their ages ranged between 5 and 15 at the beginning of the trial. At week 12, patients taking deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared to those taking a placebo. By week 52, patients in the Emflaza group maintained an overall stability in average muscle strength.

Though side effects caused by the drug have been reduced over the years, they remain similar to those resulting from other corticosteroids. More importantly, the drug results in less severe side effects typically experienced when using corticosteroids, scuh as bone mass loss, weight gain, behavioral issues, and glucose intolerance. It can also lead to a reduction in the density of the bones.