Life changing medicine to be listed on the PBS, providing hope for those with Spinal Muscular Atrophy

On Friday 22 April, the Pharmaceutical Benefits Advisory Committee (PBAC) gave a positive recommendation for nusinersen, a new medication available for people with Spinal Muscular Atrophy (SMA), to be listed on the Pharmaceutical Benefits Scheme (PBS) for adults over 18 years of age. At over $100,000 a dose, the PBS listing gives new hope to those living with this progressive neuromuscular condition.

St Vincent’s Hospital Melbourne currently provides Australia’s largest adult nusinersen service, with 12 adults now receiving treatment and another five due to commence soon.

SMA affects approximately one in 10,000 live births in Australia, which is typically diagnosed in childhood and in more severe cases this translates to inability to walk, loss of arm and hand function, reliance on mechanical ventilation and shortened life expectancy.

While there is no cure for SMA, patients living with this condition wish to maintain their independence for as long as possible and nusinersen could make that possible. 

At St Vincent’s, this service takes an interdisciplinary approach with patients receiving care from the Neurology and Interventional Radiology teams. Neurologist, Associate Professor Lauren Sanders who coordinates the service, believes that this service will play a pivotal role in improving the quality of life for patients living with SMA. "The positive recommendation from the PBAC follows years of advocating and I’m really proud of the role St Vincent’s has played in this. We have worked closely with the SMA community to understand what is important and it’s really exciting to think we can now achieve some of those goals,” said A/Prof Sanders. 

Real life impact

Josh, 28, receives nusinersen treatment at St Vincent’s and this treatment is important to patients like Josh because it gives them hope in finding stability in their condition. “I think the biggest thing is that I can get through the whole day and not be tired at the end of the day, which is something I wouldn’t have been able to do before,” said Josh. 

Other reported benefits of nusinersen are improvements in fine motor control, core stability and endurance. Small gains such as these can give patients real life impact in big ways, such as enabling them to remain in their current work or to have the endurance to eat a meal without feeling fatigued. “I was so excited and relieved when I could finally come in for my first dose,” said Josh, who has just received his fourth dose at St Vincent’s.

Paula, 40, believes nusinersen has improved her life and the lives of those around her. Once Paula’s SMA progressed, she lost a lot of independence and started to rely on other people, which was a challenge for her. “Within four treatments, I felt stronger, happier, more motivated and could do more on my own. My quality of life has improved and my future looks a lot brighter,” said Paula.