A Northbrook company said it has won fast-track status for approval of a drug that could become the first in the United States to treat Duchenne muscular dystrophy, a fatal muscle disease found most often in boys.
Privately held Marathon Pharmaceuticals said it hopes to win federal approval for deflazacort by 2016.
About 20,000 boys and young men in the U.S. live with Duchenne, a progressive disease that's passed by mothers to sons but rarely to daughters, though the daughters can become carriers. It is characterized by a progressive loss of muscle strength, with many losing the ability to walk in their teens. Patients often die from respiratory or heart failure.
Deflazacort, a steroid, has been shown to prolong lives as well as extend the ability to walk and breathe. But it is not a cure. It has been available in other countries such as Canada, Mexico and the United Kingdom for decades.
U.S. Food and Drug Administration approval is eagerly awaited by people such as Ellen Wagner, whose son, Tim, was diagnosed at 2 with Duchenne. The McHenry mom began ordering deflazacort from the United Kingdom after the physician treating her son wrote a prescription. Its use in the U.S. is legal because there is no other approved treatment for Duchenne in the country.
"From a parent's perspective, you've just been given a diagnosis that's life-threatening, it's fatal. There's nothing you can do for him," Wagner said. "And by the way there's no drug that's sold in the United States.
"No parent should have to go through that."
Because it's not approved in the U.S., insurance doesn't cover the drug. Parents pay about $125 a month for imported deflazacort.
After several runarounds with U.S. customs and too many scares that she wouldn't receive the shipment in time, Wagner said she switched Tim, now 16, to prednisone a few years ago, an off-label treatment. She said Tim experiences more negative side effects such as weight gain with the drug, a steroid. But at least she isn't tortured waiting for every order.
"It's a scary thought when you think you're going to run out of the drug. What are you going to do?" she said. "If we were able to get it in the U.S., I do think we would switch him back."
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Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016
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