FDA Approves Duchenne Muscular Dystrophy Drug

Scientists in Australia had originally developed Eteplirsen®, a drug that treats a rare and fatal muscle-wasting disease affecting one in 3,500 boys worldwide.

AsianScientist (Sep. 29, 2016) – A drug for the treatment of Duchenne muscular dystrophy (DMD) originally developed at the University of Western Australia has been approved by the US Food and Drug Administration.

As the first treatment for DMD that addresses the cause of the disease, Eteplirsen® offers new hope for sufferers of this rare and fatal muscle-wasting disease affecting one in 3,500 boys worldwide. Without treatment, affected boys are usually confined to a wheelchair before age 12 and succumb to the disease by age 30.

The FDA approval was granted to Sarepta Therapeutics, a US biotechnology company that licensed the rights to develop the drug. The patented treatment should be useful for DMD patients with a mutation in a particular region of the dystrophin gene.

Professors Steve Wilton and Sue Fletcher developed the treatment at UWA’s Center for Neuromuscular and Neurological Disorders and the Western Australian Neuroscience Research Institute and have been working with Sarepta to get the drug through clinical trials.

The approval was a fairly controversial one, following months of debate about whether Sarepta had provided enough evidence to demonstrate whether Eteplirsen® had a meaningful impact on patients.


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