The LOI is a critical step in ensuring patients living with PNH or aHUS have public access to Ultomiris, the first and only long-acting C5 complement inhibitor
MISSISSAUGA, ON,
PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterised by red blood cell destruction within blood vessels (also known as intravascular haemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots). 1-3
aHUS is a progressive, chronic condition with relapses. It is a type of thrombotic microangiopathy, which is a group of severe and potentially life-threatening rare disorders that cause blood clots and damage to the walls of the smallest blood vessels. The blood clots can cause injury to organs that may lead to organ failure and death.6-9
"In many countries around the world, ravulizumab is first-line therapy for most patients with PNH. We're pleased to see that the path is now clear for patients in
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"aHUS Canada is pleased that the pCPA has come to an agreement to make ravulizumab publicly available to aHUS patients in
Gaby Bourbara, General Manager of
PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterised by red blood cell destruction within blood vessels (also known as intravascular haemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots). 1-3
PNH is caused by an acquired genetic mutation that may happen any time after birth and results in the production of abnormal blood cells that are missing important protective blood cell surface proteins. These missing proteins enable the complement system, which is part of the immune system and is essential to the body's defence against infection, to 'attack' and destroy or activate these abnormal blood cells.1 Living with PNH can be debilitating, and signs and symptoms may include blood clots, abdominal pain, difficulty swallowing, erectile dysfunction, shortness of breath, excessive fatigue, anaemia and dark-coloured urine.1,4,5
aHUS is a type of thrombotic microangiopathy, which is a group of severe and potentially life-threatening rare disorders that cause blood clots and damage to the walls of the smallest blood vessels. The blood clots can cause injury to organs, that may lead to organ failure and death.6-9 Signs, symptoms and complications of TMA include organ damage, such as to the kidneys, heart, brain, and/or other organs; shortness of breath; high blood pressure; thrombosis (blood clots); low platelet count; anaemia; fatigue, confusion and bruising.6,8-14
aHUS is a progressive, chronic condition with relapses. It is caused by a combination of genetic and/or environmental factors resulting in dysregulation of the complement system, which is part of the body's immune system and essential to the body's defence against infection. It may appear in the presence or absence of a "trigger" or co-existing condition.15-19
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. 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. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose.
Ultomiris is approved in the US, EU and
Ultomiris is also approved in the US, EU and
Additionally, Ultomiris is approved in the US, EU and
Further, Ultomiris is approved in the EU and
As part of a broad development programme, Ultomiris is being assessed for the treatment of additional haematology and neurology indications.
Alexion, AstraZeneca Rare Disease, is the group within
- Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-2811.
- Griffin M, Hillmen P, Munir T, et al. Significant hemolysis is not required for thrombosis in paroxysmal nocturnal hemoglobinuria. Haematologica. 2019;104(3):e94-e96.
- Hillmen P., et al. The Complement Inhibitor Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2006;355(12):1233-43.
- Hillmen, P., et al. Effect of the complement inhibitor eculizumab on thromboembolism on patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123-4128.
Kulasekararaj, AG , Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540–549.- Arnold D, Patriquin C, Nazy I. Thrombotic microangiopathies: a general approach to diagnosis and management.
Canadian Medical Association Journal . 2017. 189 (4):153-159. - Blasco M, Guillén-Olmos E, et al. Complement Mediated Endothelial Damage in Thrombotic Microangiopathies. Frontiers in Medicine. 2022. 9:1-12.
- Barbour T, John S, Cohney S, et al. Thrombotic microangiopathy and associated renal disorders. Nephrology Dialysis Transplantation. 2012. 27(7):2673–2685.
- Blasco M, Guillèn E, et. al. Thrombotic microangiopathies assessment: mind the complement.
Clinical Kidney Journal . 2020. 14(4):1055-1066. - Greenbaum L, Licht C, Nikolaou V, et al. Functional Assessment of Fatigue and Other Patient-Reported Outcomes in Patients Enrolled in the Global aHUS Registry. Kidney International Reports. 2020. 5:1161-1171.
- Timmermans S, Abdul-Hamid M, et al. Patients with hypertension-associated thrombotic microangiopathy may present with complement abnormalities.
Kidney International . 2017. 91(6): 1420-1425. - Thomas M,
Robinson S ,Scully M. How we manage thrombotic microangiopathies in pregnancy.British Journal of Haematology . 2016. 173(6) 821-830. - Meri S. Complement activation in diseases presenting with thrombotic microangiopathy.
European Journal of Internal Medicine . 2013. 24(6):496-502. - Sridharan M, Abraham R, Amer H, et al. Atypical Hemolytic Uremic Syndrome-Clinical Presentation, Treatment, and Short Term Outcomes: The Mayo Clinic Experience.
- Jokiranta, TS. HUS and atypical HUS. Clinical Platelet Disorders. 2017. 129(21).
- Asif, A., Nayer, A. & Haas, C.S. Atypical hemolytic uremic syndrome in the setting of complement-amplifying conditions: case reports and a review of the evidence for treatment with eculizumab. J Nephrol. 2017. 30, 347–362.
- Afshar-Kharghan, V. Atypical hemolytic uremic syndrome. Hematology Am Soc Hematol Educ Program. 2016 (1): 217–225.
- Brambilla, M, Ardissino, G, Paglialonga, F, et al. Haemoglobinuria for the early identification of aHUS relapse: data from the ItalKId-HUS Network. J Nephrol. 2022. 35, 279–284.
- Merle, N. S., et al. Complement System Part II: Role in immunity. Frontiers of Immunology. 2015. 6:257.
SOURCE Alexion AstraZeneca Rare Disease
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