Marathon to Charge $87000 for Duchenne Drug That Costs $1600 Overseas

Feb 12, 2017, 00:55
Marathon to Charge $87000 for Duchenne Drug That Costs $1600 Overseas

It affects one in every 3600 male kids worldwide. So why the huge price for deflazacort in the U.S.? It can be imported for approximately $1,200 annually. It has priced the new product at $89,000 per year, ahead of any discounts or rebates, while prednisone can be had for as little as $50 per month.

The drug was never sold in the US because companies didn't think it would be profitable enough, The Wall Street Journal reported, but some families have obtained the drug from online pharmacies overseas to help improve muscle strength for boys at least for a while.

Illinois-based Marathon Pharmaceuticals will market deflazacort under the brand name Emflaza for a topline price of $89,000, 70 times higher than its price overseas, according to the Wall Street Journal.

A physical therapist treats a patient with DMD.

The news of deflazacort's approval, as well as its high sticker price, has parents of DMD children mostly excited. Under that status, Marathon has exclusive rights to sell deflazacort in the United States for seven years.

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Since so few Americans are affected by the disease, only around 15,000, the drug was approved under an "orphan drug" status, which is a special status given to drugs that treat rare diseases or conditions. 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. It improves muscle strength. USA patients have been importing it since the 1990s after clinical studies showed it could reduce inflammation with less unwanted side effects than another steroid.

At week 12, patients receiving deflazacort had improvements in muscle strength compared with those taking a placebo, which was maintained at week 52. Some studies have shown that patients on deflazacort tend to lose the ability to walk later that the ones who are not treated.

The adverse events (AEs) associated with deflazacort are similar to those caused by other corticosteroids. The most common side effects include facial puffiness, weight gain, upper respiratory tract infection, cough, increased appetite and hair growth, and excessive fat around the stomach.

Less common side effects include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk for gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones, and vision problems.