First targeted treatment on the PBS for rare, unpredictable and debilitating autoimmune disease, NMOSD

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.^4,5Most 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.

– ENDS –

No compensation was provided to Professor Michael Barnett in relation to this announcement and the opinions expressed are his own. He has been briefed by Alexion on the approved use of this product.

Professor Barnett has received consultancy fees/honorarium for speaking engagements, was an Advisory Board member, and has received institutional research funding from Alexion.

THERAPEUTIC INDICATION(S): For the treatment of adult patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) who are anti-aquaporin 4 (AQP4) antibody-positive. Ultomiris is not intended for the acute treatment of a NMOSD relapse. Dose and Administration: Loading dose and minimum infusion duration time: 2400 mg over 45 min (≥ 40 to < 60 kg), 2700 mg over 35 min (≥ 60 to < 100 kg), 3000 mg over 25 min (≥ 100 kg) followed by maintenance dose and minimum infusion duration time (every 8 wks, starting 2 wks after loading dose): 3000 mg over 55 min (≥ 40 to < 60 kg), 3300 mg over 40 min (≥ 60 to < 100 kg), 3600 mg over 30 min (≥ 100 kg); for pts switching from Soliris^® to Ultomiris, administer loading dose of Ultomiris 2 wks after last Soliris maintenance infusion or 1 week after the last Soliris induction infusion; dilute to 50 mg/mL with 0.9% sodium chloride; administer intravenously through 0.2 μm filter. Patients should be monitored post infusion for signs or symptoms of an infusion-related reaction. Supplemental dose of Ultomiris in the setting of IVIg, PE or PP (see full PI). CONTRAINDICATIONS: hypersensitivity to ravulizumab rch or excipients; unresolved Neisseria meningitidis infection; unvaccinated against Neisseria meningitidis (unless patients receive appropriate prophylactic antibiotics until 2 wks after vaccination). SPECIAL WARNINGS AND PRECAUTIONS FOR USE: Serious Meningococcal Infection: refer to Boxed Warning above; increased meningococcal (N. meningitidis) infection susceptibility; cases of serious or fatal meningococcal infections/sepsis have been reported Immunisation: ensure immunisation currency; vaccination may further activate complement, closely monitor for disease symptoms following vaccination; vaccination may not be sufficient to prevent meningococcal infection, prescriber and patient should discuss the potential role of ongoing preventative antibacterials, monitor for signs of infection, including fever, headache +/- stiff neck, light sensitivity; patients below the age of 18 years old must be vaccinated against Haemophilus influenzae and pneumococcal infections. Other Systemic Infections: administer with caution in patients with active systemic infections; increased susceptibility to infections, especially infections caused by Neisseria species, including gonococcal infections. Infusion Reactions: administration of Ultomiris may result in infusion reactions and allergic/hypersensitivity reactions (including anaphylaxis); in an infusion reaction, infusion of ravulizumab should be interrupted and supportive measures instituted if signs of cardiovascular instability or respiratory compromise occur.  Monitoring after treatment discontinuation: if treatment with Ultomiris is discontinued, patients should be monitored for symptoms of the underlying disease. If symptoms of NMOSD occur after discontinuation, consider restarting the treatment. Use in Pregnancy-Category B2: ensure adequate contraception for women of childbearing potential during and up to 8 months post-treatment. Use in Lactation: discontinue breastfeeding during and up to 8 months post-treatment. Adverse Effects: Meningococcal infection/sepsis, upper respiratory tract infection, urinary tract infection, gastrointestinal infection, pneumonia, cough, hypersensitivity (including anaphylaxis, infusion reaction (discontinue if severe) headache, migraine, anxiety,  diarrhoea, constipation, nausea, vomiting, gastroesophageal reflux disease, pyrexia, chills, pain in extremity, abdominal pain, dizziness, arthralgia, myalgia, back pain, muscle spasms, anaemia, peripheral oedema, hypertension, fatigue, hypokalemia, contusion, dyspnea, alopecia, dry skin, rash, urticaria, lymphadenopathy, malaise, non-cardiac chest pain, vaccination site pain, COVID-19, intervertebral discitis (see full PI). Date Revised: January 2024. REF: ULT100/NMOSD /PI/22JAN2024

For healthcare professionals, please refer to the full Ultomiris Product Information before prescribing, which can be accessed at: Ultomiris Product Information.

For more information about Ultomiris, the Consumer Medicine Information can be found here: Ultomiris Consumer Medicine Information.

Notes

NMOSD

NMOSD is a rare disease in which the immune system is inappropriately activated to target healthy tissues and cells in the central nervous system (CNS).⁴ Australian data suggest most people (~90%) with NMOSD are anti-aquaporin-4 (AQP4) antibody-positive.⁹ This binding can inappropriately activate the complement system, which is part of the immune system and is essential to the body’s defence against infection, to destroy cells in the optic nerve, spinal cord and brain.^5,10

It most commonly affects women and begins in the mid-30s. Men and children may also develop NMOSD, but it is even more rare.^11-14 People with NMOSD may experience vision problems, intense pain, loss of bladder/bowel function, abnormal skin sensations (e.g. tingling, prickling or sensitivity to heat/cold), and impact on coordination and/or movement.^5,6Most people living with NMOSD experience unpredictable relapses, also known as attacks. Each relapse can result in cumulative disability including vision loss, paralysis, and sometimes premature death.^5,6 NMOSD is a distinct disease from other CNS diseases, including multiple sclerosis. The journey to diagnosis can be long, with the disease sometimes misdiagnosed.^4,15

Ultomiris

Ultomiris belongs to a class of medicines called monoclonal antibodies, that attach to a specific target in the body. Ravulizumab rch, the active ingredient in Ultomiris, has been designed to attach to the C5 complement protein, which is a part of the body’s defence system called the ‘complement system’.^2,3

Ultomiris is approved for use in Australia for the treatment of autoimmune diseases including Paroxysmal Nocturnal Haemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS), generalised Myasthenia Gravis (gMG) and Neuromyelitis Optica Spectrum Disorder (NMOSD).^2,3

Alexion

Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. Please visit https://alexion.com/worldwide/Australia.

AstraZeneca

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca For more information, please visit www.astrazeneca.com.au

Alexion Pharmaceuticals Australasia Pty Ltd. Macquarie Park, NSW 2113. Medical enquiries: 1800 788 189. March 2025. AU/ULT-N/0021.

Contacts

Nicki Sambuco M: +61 452 446 084 E: nicki@senateshj.com.au.

References

1. Australian Government. Department of Health and Aged Care. The Pharmaceutical Benefits Scheme (PBS) [Online]. Available at: https://www.pbs.gov.au/. Accessed March 2025.
2. Alexion. 2024. Ultomiris Consumer Medicine Information. [Online]. Available at: https://www.ebs.tga.gov.au/ebs/picmi/picmirepository.nsf/pdf?OpenAgent&id=CP-2021-CMI-01355-1Accessed March 2025.
3. ULTOMIRIS^® Australian Approved Product Information. Available at: https://www.ebs.tga.gov.au/ebs/picmi/picmirepository.nsf/pdf?OpenAgent&id=CP-2021-PI-01352-1Accessed March 2025.
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5. Wingerchuk DM, et al. The Spectrum of Neuromyelitis Optica. Lancet Neurol. 2007;6(9):805-15.
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8. Australian Bureau of Statistics. Population clock and pyramid. [Online] Available at: https://www.abs.gov.au/statistics/people/population/population-clock-pyramid Accessed March 2025
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10. Yick LW, et al. Aquaporin-4 Autoantibodies From Neuromyelitis Optica Spectrum Disorder Patients Induce Complement-Independent Immunopathologies in Mice. Front. Immunol. 2018;9:1438.
11. Bukhari W, et al. Incidence and Prevalence of NMOSD in Australia and New Zealand. J Neurol Neurosurg Psychiatry. 2017:88(8):632-638.
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15. Mealy MA, et al. Assessment of Patients with Neuromyelitis Optica Spectrum Disorder Using the EQ-5D. Int J MS Case. 2019;21(3):129–134.