What is the innovation?

Eteplirsen® offers new hope for sufferers of Duchenne Muscular Dystrophy (DMD), a rare and fatal muscle wasting disease affecting one in 3,500 boys worldwide.

The innovation is a “genetic bandaid” that allows certain patients with DMD to make a shorter, but useful, form of dystrophin, a protein that is crucial for muscle function. Without the protein patients lose muscle function and mobility, and develop cardiac and respiratory issues, often succumbing to the disease as young adults.

Even a small amount of the shorter form of the protein can improve the mobility and quality of life of patients with DMD. Different groups of patients needs different bandaids, depending on where on the gene the bandaid is needed.

What benefits does it bring?

Eteplirsen is the first ‘disease-modifying’ treatment for DMD. Whilst not a cure it’s the first treatment that restores muscle function and delays the progression of the disease.

What commercial success and/or benefit to society has it achieved?

Eteplirsen was licensed to global biotechnology company Sarepta Therapeutics and received ‘accelerated approval’ from the US Food & Drug Administration (FDA) in Sept 2016.

It is now on the market in the USA with clinical trials underway elsewhere in the world. Similar treatments are being developed so that as many DMD patients as possible can be treated.

Sarepta reported almost US$50M of revenue for Eteplirsen in Q3 2017.

What lessons learnt can you share?

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. They are now continuing their groundbreaking research at Murdoch University.

Developing therapies takes a lot of time (we started in 2004) and a lot of money (Sarepta spent hundreds of millions of dollars funding the clinical trials), so partnering early with companies who know the business is crucial.

In this case filing a patent application was critical; without it the university wouldn’t have been able to attract a partner to fund the development of the treatment.