Max and Austin Leclaire have lived a lot of hard days together. The brothers have Duchenne muscular dystrophy, a rare degenerative disease that wastes their muscles and will likely take their lives at an early age.
Over the last few years, they found a glimmer of hope in an experimental drug, called eteplirsen. Max was able to get on the drug in 2011. Austin started on it three years later in another clinical trial. Both boys believe it has helped slow their physical decline. And Max has remained able to walk, although not easily, beyond the age when boys with Duchenne usually lose the use of their legs.
The company that developed the drug, Sarepta Therapeutics, has been pushing hard for federal approval to take it to market. It doesn’t have a lot of hard data that it works; in part because Duchenne muscular dystrophy is so rare, Sarepta presented data on just a handful of patients to the Food and Drug Administration.
Scientists at the FDA were deeply divided, but on Monday, the agency finally approved the drug. It’ll be available within a few months, at an estimated price of $300,000 a year.
The approval pleased Max, 14, and Austin, 17. And not just because they hope to keep taking the drug.
“It was a pretty big relief,” Austin said, “but I was more worried about the other kids, that if it didn’t get approved, they wouldn’t have a chance.”
Muscular dystrophy problem
I am very happy that these children get a chance – at a drug that has been safety tested for 2 yrs +. what have they got to lose ? I am glad that Dr. Woodcock is listening to pts. now. I wish she and Dr.s’ Hamburg and Pazdur at the FDA had been listening to pt.’s like us back in 2010 when they rejected early approval for several very successful , life saving drugs . Drugs like kadcyla- that saved my wifes’ life. Maybe our Pt. Rights protest helped soften the FDA up – I think so . Go read about my wife, Lorraine Heidke Mccartin – her Right To Try story on the Goldwaterinstitute.org Right To Try webpage. She is still cancer free 5 yrs and counting. No thanks to the FDA of 2009 -2014- flexing their regulatory muscles with Pharma – and punishing dying pts in the process. Here’s to more early approval / compassionate use.
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