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), which can attack optic nerves and result in vision loss.
A media release said the treatment was listed on the Pharmaceutical Benefits Scheme (PBS) on 1 April 2025.
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.
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.
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. Around 290 Australians are estimated to be living with NMOSD.
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.”
The release said that Ultomiris belonged 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.
Mr 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.”
