gamesindustryblog.com
gamesindustryblog.com February 21, 2017


FDA approves costly drug to fight Duchenne Muscular Dystrophy

12 February 2017, 12:55 | Elizabeth Houston

Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system

Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system

Until now, the drug was not sold in America because no company thought it was worth the effort. It has been available for years in other countries, and the family of the individuals with Duchenne Muscular Dystrophy. Those with the disease usually lose the ability to walk in their teens and have a life expectancy around 25 to 30 years.

Now, with an FDA-approved med on the market-the only one green-lighted to treat all DMD patients age 5 and older-the import option is off the table for patients, and Marathon is slapping an $89,000-per-year list price on the med. With the orphan designation, the company is getting seven years of exclusive rights to sell the drug in the U.S.

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.

A voucher known as a priority review was given to the company, which essentially is equal to a ticket that can be used to get a drug in the future reviewed faster by regulators, or can be sold for hundreds of millions of dollars to another pharmaceutical company.

Ryanair 3Q profits dip, but full-year figures unchanged
Ryanair has said it had to cut its fares more than expected during the last three months of 2016, which pulled down profits . It said these were causing boarding delays as punctuality fell from 90% previous year to 88% over the past nine months.

Ghias also says that that after rebates and discounts, the price will drop to $54,000, and with insurance and financial programs most patients will pay "zero to low out-of-pocket expense, " according to the Washington Post. 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. In general, the muscle strength was maintained among those taking the drug until the end of the 52-week study.

The most common side effects include facial puffiness (cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, and central obesity.

"This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy", said Billy Dunn, director of the Division of Neurology Products at the FDA.

Other less-common AEs include problems with endocrine function, increased susceptibility to infection, hypertension, the risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decreased bone density, and vision problems, such as cataracts. Patients have been importing the drug since the 90s, but now they will have to pay 50 to 70 times what they used to pay if they want to get it in the U.S.



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