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article imageFDA approves $89,000 drug to treat Duchenne's Muscular dystrophy

By Karen Graham     Feb 10, 2017 in Health
The U.S. Food and Drug Administration (FDA) on Thursday gave approval to a drug manufactured by Marathon Pharmaceuticals to treat Duchenne's muscular dystrophy (DMD), a devastating muscle disease that usually affects boys.
In a press release dated February 9, 2017, the FDA approved Marathon's Emflaza tablets and oral suspensions as a treatment for patients ages five and older with Duchenne's muscular dystrophy (DMD), a rare genetic disorder that causes a progressive deterioration of muscles and progressive weakness.
Emflaza is a corticosteroid, much like prednisone, that works by decreasing inflammation and reducing the activity of the immune system. There is just one really big problem with the drug - It will cost patients $89,000 a year, an exorbitant price when compared to its cost in other countries around the world.
But Marathon CEO Babar Ghias told the Washington Post that after rebates and discounts, the net price will only be $54,000.
The generic name for Emflaza is called deflazacort and like similar corticosteroids, is used to treat a variety of conditions. The most common drugs used to treat DMD are prednisone and deflazacort, although neither drug will alter the outcome of the disorder.
Both drugs are used to slow down the progression of the disease, allowing the patient to have the ability to ambulate (walk) longer and delay becoming dependent on a wheelchair. The drugs also slow down the loss of arm function and slow down the onset and progression of respiratory and cardiac problems common to the disease.
So why the huge price for deflazacort in the U.S.?
In Canada and other countries around the world, deflazacort has been used for years to treat DMD and the pills or oral suspensions range from about $1,000 to $2,000 a year for patients. And Marathon didn't invent a new drug, either. They are basically being allowed to make the same drug sold in other countries, but they have given it a new name.
Believe it or not, but it is the law. Because this particular steroid has never been approved for use in the United States, the FDA considers it a new drug. This means that Marathon had to go through the process of getting it approved as a new drug and they were also able to reap the benefit of laws Congress has passed to encourage drug companies to develop new medicines for rare diseases.
Those legal benefits include having a monopoly on the drug under the Orphan Drug Act and the privilege of getting a rare disease priority review voucher that allows a company to get a sped-up FDA review for another drug they may come up with. The vouchers can be sold to other pharmaceutical companies, commanding a huge price.
This is the best way to get pharmaceutical companies to come up with new drugs, and the public is going to have to pay for the research and development behind those drugs, according to the FDA. But deflazacort is a generic steroid, and should not be treated as a "new" drug.
Forbes says the price Marathon is asking patients to pay for the drug is "absurd." And more to the point, "It is not right." It is nothing more than price-gouging all over again, just like the EpiPen and other drugs that have been in the news recently. And people should not take this sitting down.
More about DMD, Marathon Pharmaceuticals, corticosteroids, Emflaza, Expensive
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