Ultomiris is the first approved, long-acting complement C5 inhibitor for PNH administered every eight weeks 1
PNH is an extremely rare, life-threatening disease that can damage vital organs and, if left untreated, can lead to premature death 2,3,4
Sydney, Australia, 1 March 2022 – Alexion Pharmaceuticals Australasia Pty Ltd, AstraZeneca's Rare Disease group, welcomes the listing of Ultomiris (ravulizumab-rmc) on the Pharmaceutical Benefits Scheme (PBS) for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH).1 Australians living with this ultra-rare, life-threatening disease will now have access to this first and only long-acting complement C5 inhibitor administered every eight weeks (following induction dose and first maintenance dose).1
Chhean Khoun, President of the Australian PNH Support Group (PNHSAA) said: "This is a great day for Australians who live with PNH. It means so much to the individuals and families who live with this chronic condition and have advocated for access to this new treatment option. We are grateful to the government for recognising the value of the medicine and how it can relieve the burden for anyone with PNH."
PNH is an ultra-rare, chronic blood disorder where the body destroys its own red blood cells. The body's platelets are more active than normal increasing the risk of blood clots forming which can lead to organ damage and premature death.2,3,4 Patients with PNH can experience debilitating symptoms, including pain, fatigue and shortness of breath, preventing them from being able to work and socialise.5 PNH can strike men and women of all ages without warning, with an average age of onset in the early 30s.6,7 The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis – in addition to appropriate treatment – is critical to improving patient outcomes.5,8
For the last decade, Australians with PNH were granted access to Soliris (eculizumab-rch) under the Life Saving Drugs Program. With Ultomiris now listed on the PBS, and Soliris also moving from the Life Saving Drugs Program to the PBS, patients in consultation with their treating physician can discuss the most appropriate treatment option to best manage their health and lifestyle.
Haematologist, Professor Jeff Szer said: "As someone committed to the management of patients with ultra-rare blood diseases, I welcome this new listing of Ultomiris for the life-threatening disorder PNH. It expands possibilities in so many ways to the advantage of the patient who can now be secure in the knowledge that they have a safe, effective treatment that helps them lead a near normal life. The movement to a PBS listing will make therapy for this disease more accessible to many patients."
Soliris and Ultomiris are complement inhibitors that work to inhibit the C5 protein in the terminal complement cascade, a part of the body's immune system.1,9
Sara Trafford Jones, General Manager Alexion Australia also welcomed the listing. "Managing a life-long, rare disease can be a heavy burden for patients and families. Being able to provide access to a new treatment option is part of our deep commitment to developing and delivering innovative therapies for people and families with rare diseases."
Both Soliris and Ultomiris will be available to patients on the PBS from 1 March 2022.
PNH
PNH is a serious ultra-rare blood disorder with devastating consequences. It is characterised by the destruction of red blood cells, which is also referred to as haemolysis.2,3 PNH occurs when the complement system – a part of the body's immune system – over-responds, leading the body to attack its own red blood cells. PNH often goes unrecognised, with delays in diagnosis from one to more than five years.10 Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-coloured urine and anaemia.6,11 The most devastating consequence of chronic haemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death. 4 The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis – in addition to appropriate treatment – is critical to improving patient outcomes. 5,8
Ultomiris
Ultomiris (ravulizumab-rmc), is the first and only long-acting complement C5 inhibitor.12 The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells.13 Ultomiris is administered intravenously every 8 weeks after an induction dose and the first maintenance dose after 2 weeks.1
▼This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events at www.tga.gov.au/reporting-problems.
Please refer to the Full Ultomiris Product Information before prescribing, which can be accessed at https://secure.guildlink.com.au/gc/ws/xi/pi.cfm?product=xipultoi
Soliris
Soliris (eculizumab-rch) is a first-in-class complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade, a part of the immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe rare and ultra-rare disorders.13
PBS Information: Ultomiris® is listed on the PBS as a Section 100 item for the treatment of adults with Paroxysmal Nocturnal Haemoglobinuria (PNH). Refer to PBS Schedule for full authority information.
▼This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events at www.tga.gov.au/reporting-problems.
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.
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. F
