SPINRAZA becomes first available treatment on the Pharmaceutical Benefits Scheme for adults living with Spinal Muscular Atrophy

Sydney, Australia – 1 August 2022.

From 1 August 2022, adults (at least 19 years of age or older) with 5q Spinal Muscular Atrophy (SMA) and symptom onset prior to their 19th birthday will be able to access SPINRAZA® (nusinersen) on the Pharmaceutical Benefits Scheme (PBS),1 the first and only reimbursed treatment for this patient population in Australia.2

SMA is a rare genetic disease caused by a shortage of a protein called survival motor neuron (SMN). This results in the loss of nerve cells in the spine, leading to progressive weakness of the muscles in the shoulders, hips, thighs, and upper back. For adults living with SMA the level of muscle weakness can be significant, including paralysis and difficulty with the most basic functions of life, like breathing and swallowing.3,4

SPINRAZA is a type of medicine called an anti-sense oligonucleotide that works by making the body produce more SMN protein in a type of SMA known as 5q SMA. 3

SMA occurs in approximately one in 6,000 – 11,000 births.5,6 Currently, there are approximately 650 to 800 Australians living with SMA.7

Professor Matthew Kiernan, Bushell Chair of Neurology at Royal Prince Alfred Hospital and Co-Director of the Brain and Mind Centre, University of Sydney said SMA has a significant impact on the day to day lives of adults with the disease. “For adults with SMA this results in a loss of functional ability over time, with an increased risk of respiratory infections and increased hospital admissions. For many, it becomes a progressive battle against time to preserve quality of life and independence which can be distressing for themselves, their carers, and family members,” Professor Kiernan said.

SPINRAZA has been available on the PBS since June 2018 for children with SMA who present with symptoms prior to three years of age, and since 2020 for infants with pre-symptomatic SMA.8,9 “Until now there have been no treatment options available for adults living with SMA. This PBS listing is welcome news and means that Australians living with SMA now have access to treatment regardless of age,” Julie Cini, Spinal Muscular Atrophy Australia spokesperson said. “This was a highly anticipated decision the SMA community has been waiting for.”

David Henderson, Managing Director, Biogen ANZ said, “At Biogen ANZ, we are proud of our continued commitment to both adults and children living with SMA. We understand the impact SMA has not only on the individual but on their families and those who care for them. This announcement today exemplifies why the connection between science and humanity is so important, as we continue to invest in research and support for the SMA community.”

PBS Information:
SPINRAZA® is listed on the Pharmaceutical Benefits Scheme (PBS) as a Section 100 Item. For use in patients with spinal muscular atrophy (SMA) who meet certain criteria.Refer to PBS Schedule for full Authority Information.

For further information, please see Consumer Medicine Information:

For further information please contact Fiona Tigar +61 400 741 286.


1.     Australian Government Department of Health. The Pharmaceutical Benefits Scheme. Recommendations made by PBAC – March 2022. March 2022 - PBAC Outcomes. Available at: https://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/pbac-outcomes/recommendations-made-by-the-pbac-march-2022 [Last accessed 17 June 2022].
2.     Australian Government, Department of Health, Therapeutic Goods Administration (2017). Register of Therapeutic Goods. [online] Available at: https://www.tga.gov.au/prescription-medicines-registration-new-chemical-entities-australia-2017 [Last Accessed 17 June 2022].
3.     Biogen SPINRAZA Consumer Medicine Information (2021). https://www.guildlink.com.au/gc/ws/bd/rss.cfm?product=bdcspinr21117. [Last accessed 14 July 2022]
4.     National Center for Advancing Translational Science. Genetic and Rare Diseases Information Centre. Available at: https://rarediseases.info.nih.gov/diseases/7674/spinal-muscular-atrophy [Last accessed 17 June 2022].
5.     Lin C-W et al. Delay in Diagnosis of Spinal Muscular Atrophy: A Systematic Literature Review. Pediatric Neurology 53 (2015) 293e300.
6.     D’Amico A et al. Spinal muscular atrophy. Orphanet Journal of Rare Diseases 2011, 6:71 http://www.ojrd.com/content/6/1/71 7.
7.     Biogen data on file
8.     Australian Government Department of Health. Media Release. 15 November 2020. Life-saving Spinal Muscular Atrophy medicine to be made available to Australian babies. Available at https://www.health.gov.au/ministers/the-hon-greg-hunt-mp/media/life-saving-spinal-muscular-atrophy-medicine-to-be-made-available-to-australian-babies#:~:text=Spinraza%20is%20the%20first%20and,Benefits%20Advisory%20Committee%20(PBAC).[Last accessed 17 June 2022].
9.     Australian Government Department of Health. Life-saving Spinal Muscular Atrophy medicine to be made available to Australian babies. Available at: https://www.health.gov.au/ministers/the-hon-greg-hunt-mp/media/life-saving-spinal-muscular-atrophy-medicine-to-be-made-available-to-australian-babies [Last accessed 15 July 2022].