Sydney, Australia, 27 December 2023 – Alexion, AstraZeneca Rare Disease, announced that Ultomiris® (ravulizumab rch) has been listed on the Pharmaceutical Benefits Scheme (PBS) for the treatment of atypical Haemolytic Uraemic Syndrome (aHUS) for eligible patients from 1 January 2024.1,2
aHUS is a rare disorder that can occur at any age.3,4 In people with aHUS, tiny blood clots form in their small blood vessels, blocking the blood flow to important organs.3 This can lead to organ failure including kidney failure, heart attacks and other life-threatening health problems.3,5
Up to 200 Australians are estimated to be living with aHUS.6 It occurs due to dysregulation of the complement system, a part of the immune system that is important in fighting infection.4,7 In people with aHUS, overactivation of their complement system causes their red blood cells to be destroyed faster than they can be made, and their platelets to become more likely to form clots. This leads to inflammation and attacks on organs and cells, including the cells that line the blood vessels.4,5,7 In some cases, this dysregulation occurs after a ‘triggering’ event such as pregnancy, an infection, or taking certain medications, among others.4
Dr Danny Hsu, Clinical and Laboratory Haematologist at Liverpool Hospital, Conjoint Senior Lecturer (UNSW Medicine), says: “aHUS can be a devastating disease that occurs at any age. While some people may experience a loss of organ function over time, for most adult patients I have encountered, organ failure can be sudden and catastrophic.
“With the current standard of care for aHUS, the aim is to control the over activation of the complement system by a complement-inhibiting drug that is given via intravenous infusion every week for the first five weeks, then fortnightly thereafter in adults”,8 continues Dr Hsu.
“However with reimbursement now available for Ultomiris, maintenance treatment is given once every eight weeks for patients greater than 20kg, two weeks after an initial loading dose, helping reduce the infusion frequency and treatment burden for patients and their caregivers.” *
Nicole Gaupset, General Manager Alexion Australia, has welcomed the listing of Ultomiris on the PBS for aHUS. “At Alexion we are committed to continuous innovation so people with complement-mediated diseases can live their best lives. It has been nearly a decade since our first treatment for this devastating condition was made available in Australia and we are delighted to see the Australian Government continuing to invest in improving health and quality of life outcomes for Australians living with rare diseases including aHUS through the PBS.”
▼ This medicine is subject to additional monitoring due to approval of an extension of indications. This will allow quick identification of new safety information. Consumers can help by reporting any side effects directly to their doctor, or directly at www.tga.gov.au/reporting-problems. See the full CMI for further details. Healthcare professionals are asked to report any adverse events directly at www.tga.gov.au/reporting-problems.
You can also report side effects directly to Alexion at https://contactazmedical.astrazeneca.com or via 1800 180 170
PBS Information: From 1 January 2024 - Ultomiris is listed on the PBS as a Section 100 item for the treatment of Paroxysmal Nocturnal Haemoglobinuria and atypical Haemolytic Uraemic Syndrome. Refer to PBS Schedule for full authority information. Ultomiris is not listed on the PBS for the treatment of adult patients with generalised Myasthenia Gravis (gMG). |
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No compensation was provided to Dr Hsu in relation to this announcement and the opinions expressed are his own. Dr Hsu has been briefed by Alexion on the approved use of this product.
Dr Hsu has received consultancy fees/honorarium for speaking engagements, is an Advisory Board member and has received research funding from Alexion.
Media Contacts
Nicki Sambuco M: +61 452 446 084 E: nicki@senateshj.com.au
* Please see prescribing information for further detail regarding weight-based treatment phasing and dosing.
About Ultomiris
Ultomiris® (ravulizumab rch) belongs to a class of medicines called monoclonal antibodies that attach to a specific target in the body. Ultomiris has been designed to attach to the C5 complement protein, which is a part of the body’s immune defence system called the ‘complement system’.2
In Australia, Ultomiris is approved for use in the treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS) and generalized Myasthenia Gravis (gMG) in adults.2
Minimum Prescribing Information
WARNING: SERIOUS MENINGOCOCCAL INFECTION. Life-threatening meningococcal infections/sepsis have occurred in patients treated with Ultomiris®. Meningococcal infection may become rapidly life-threatening or fatal if not recognised and treated early. Refer to the most current edition of the Australian Immunisation Handbook for meningococcal vaccination guidelines. Immunise patients with meningococcal vaccines at least 2 weeks prior to administering the first dose of Ultomiris, unless the risks of delaying Ultomiris therapy outweigh the risk of developing a meningococcal infection. Vaccination reduces, but does not eliminate, the risk of meningococcal infections. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected.
aHUS
THERAPEUTIC INDICATION(S): The treatment of patients with Atypical Haemolytic Uraemic Syndrome (aHUS). Dose and Administration: Loading dose and minimum infusion duration time: 600 mg over 85 min (≥ 5 to < 10 kg), 600 mg over 45 min (≥ 10 to < 20 kg), 900 mg over 35 min (≥ 20 to < 30 kg), 1200 mg over 31 min (≥ 30 to < 40 kg) 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): 300 mg over 45 min (≥ 5 to < 10 kg), 600 mg over 45 min (≥ 10 to < 20 kg), 2100 mg over 75 min (≥ 20 to < 30 kg), 2700 mg over 65 min (≥ 30 to < 40 kg), 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. In aHUS, Ultomiris treatment should be a minimum duration of 6 months. 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, 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 closely monitored for signs and symptoms of thrombotic microangiopathy (TMA) on an on-going basis. Monitoring may be insufficient to predict or prevent TMA complications. If TMA complications occur after discontinuation, re-initiation of treatment should be considered (see full PI). 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, diarrhoea, constipation, nausea, vomiting, pyrexia, 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 (see full PI). Date Revised: July 2023. REF: ULT100/aHUS/PI/19JUL2023
For more information about Ultomiris®, the Consumer Medicine Information can be found here: https://rss.medsinfo.com.au/xi/cmi.cfm?product=xicultoi
About Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines.
Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
Please visit https://alexion.com/worldwide/Australia
About AstraZeneca
AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology; Cardiovascular, Renal & Metabolism; and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide.
For more information, please visit www.astrazeneca.com.au
Alexion Pharmaceuticals Australasia Pty Ltd Level 4, 66 Talavera Road, Macquarie Park, NSW 2113 Medical enquiries: 1800 788 189. December 2023. AU/ULT-a/0033.
References
1 Australian Government. Department of Health and Aged Care. The Pharmaceutical Benefits Scheme (PBS) [Online]. Available at: https://www.pbs.gov.au/.
2 Alexion. 2023 Ultomiris® Consumer Medicine Information. [Online] Available at: https://rss.medsinfo.com.au/xi/cmi.cfm?product=xicultoi Accessed December 2023
3 National Institutes of Health (NIH). Atypical hemolytic uremic syndrome. [Online]
Available at: https://rarediseases.info.nih.gov/diseases/8702/atypical-hemolytic-uremic- syndrome Accessed December 2023.
4 Afshar-Kharghan, V. 2016. Atypical hemolytic uremic syndrome. Hematology Am Soc Hematol Educ Program. (1): 217–225.
5 Campistol JM, Arias M, Ariceta G, et al. 2013. An update for atypical haemolytic uraemic syndrome: diagnosis and treatment. Nefrologia. Jan 18;33(1): 27-45
6 Alexion. 2023. Data on file.
7 Jokiranta, TS. 2017. HUS and atypical HUS. Blood. Clinical Platelet Disorders. 129(21).
8 Alexion. 2023. Soliris Product Information (PI). [Online] Available at: https://www.ebs.tga.gov.au/ebs/picmi/picmirepository.nsf/pdf?OpenAgent&id=CP-2010- PI-02947-3&d=20231219172310101 Accessed December 2023.