Alexion, AstraZeneca Rare Disease treatment Ultomiris® (ravulizumab rch) has become the first targeted treatment option to be reimbursed for people living with Neuromyelitis Optica Spectrum Disorder (NMOSD) following its listing on the Pharmaceutical Benefits Scheme (PBS) from 1 April 2025.1-3
Ultomiris is now reimbursed for eligible Australians with anti-aquaporin 4 (AQP4) antibody-positive (Ab+) NMOSD who have experienced a recent relapse event, despite prior treatment with rituximab or who cannot tolerate rituximab.1-3
NMOSD is a rare disease in which the immune system is inappropriately activated to target healthy tissues and cells in the central nervous system, including the brain, spine and optic nerves.4,5 Most people living with NMOSD experience unpredictable attacks, known as relapses. Each relapse can result in cumulative disability including vision loss, paralysis and sometimes premature death.5,6 Around 290 Australians are estimated to be living with NMOSD.7,8
Dr Michael Barnett, Professor of Neurology at The University of Sydney and Royal Prince Alfred Hospital, said: "Nearly a third of people with NMOSD will experience a relapse despite existing standard of care therapies. Additionally, some patients are unable to tolerate or develop significant side effects from these treatments. A single relapse can lead to profound and irreversible disability, underscoring the critical need for new treatment options. Advances in our understanding of the role of the complement system in autoimmune diseases such as NMOSD – and in particular, abnormal activation of this essential component of the immune system – have led to the development of targeted treatments such as Ultomiris."
Ultomiris belongs to a class of medicines called monoclonal antibodies that attach to a specific target in the body. The active ingredient in Ultomiris is designed to attach to a part of the complement system and block the body's inflammatory response and prevent its ability to attack and destroy nerves in the eyes, brain and spinal cord, which occurs during an attack or relapse of NMOSD.2,3
Rohan Greenland, Neurological Alliance Australia Chair, said: "NMOSD affects far more than physical health – it disrupts relationships, independence, and emotional well-being. The unpredictability of relapses and their lasting impact add to the daily burden of NMOSD, making relapse prevention critical to preserving dignity and quality of life. We extend our heartfelt thanks to our community for sharing their experiences and to the government for listening and responding with this important PBS listing."
Nicole Gaupset, General Manager, Alexion, AstraZeneca Rare Disease, Australasia, said: "Alexion has been at the forefront of innovation in complement-mediated conditions like NMOSD. Alexion is delighted that the Australian Government has acknowledged the profound impact of this disease by listing Ultomiris on the PBS, ensuring much-needed access to the first targeted treatment option for people with NMOSD who experience relapse."
Treatment options should be discussed with a patient's clinician.
This medicine is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Patients can help by reporting any side effects you may get. Patients can report side effects to their doctor, or directly at https://www.tga.gov.au/reportingproblems. Healthcare professionals are asked to report any suspected adverse events at https://www.tga.gov.au/reporting-problems.
Adverse events can also be reported to Alexion at: https://contactazmedical.astrazeneca.com.
PBS Information:
From 1 April 2025: Ultomiris® is listed on the PBS Section 100 Highly Specialised Drugs program, Authority Required (Written) for the treatment of patients with Neuromyelitis Optica Spectrum Disorder (NMOSD). Refer to PBS Schedule for full authority information.
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