A new 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 (FDA).
UWA’s Deputy Vice-Chancellor Research Professor Robyn Owens said the drug Eteplirsen® offered new hope for sufferers of DMD, a rare and fatal muscle wasting disease affecting one in 3,500 boys worldwide.
“We’re thrilled that Eteplirsen has been approved,” Professor Owen said. “It’s the first treatment for DMD that addresses the cause of the disease and the hope is that it will slow its progression and keep patients mobile for longer.”
The FDA approval was granted to Sarepta Therapeutics, a US biotechnology company that licensed the rights to develop the drug and means the treatment can now be made available to DMD patients. Without treatment, affected boys are usually confined to a wheelchair before age 12 and succumb to the disease by age 30.
Professors Steve Wilton and Sue Fletcher developed the treatment at UWA’s Centre for Neuromuscular and Neurological Disorders (CNND) and the Western Australian Neuroscience Research Institute (WANRI) and have been working with Sarepta to get the drug through clinical trials.
“What Steve and Sue and the team at Sarepta have done is amazing – to think that the results in a lab notebook at UWA have gone on to be translated into a treatment for a disease where there is currently no hope is amazing,” said Professor Owens.
“It demonstrates how universities can be the source of great ideas and how partnering with experts in industry can create real impact.”
The patented treatment should be useful for DMD patients with a mutation in a particular region of the dystrophin gene and the race is on to develop similar treatments to treat more DMD patients.
Simon Handford, Associate Director Research Development and Innovation at UWA, said the technology transfer journey from laboratory to patient had been an extensive process.
“We’ve been working with Steve and Sue since 2004 and it’s so satisfying to have played a small part in the process from discovery to approval,” Mr Handford said.
“Our hope is that this inspires other researchers to think about how they are going to translate their research findings – whether in medicine, agriculture, engineering, business – and to think about partnering at an early stage”.