ALXN 1210 for Paroxysmal Nocturnal Haemoglobinuria – First Line

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Therapeutic Areas: Haematology
Year: 2017

Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra‐rare life‐threatening blood disorder. Symptoms and signs of PNH are varied and can include: fatigue; dark red/brown urine; difficulty swallowing and abdominal pain and infections and bruising. PNH is a disorder with an age‐at‐onset typically ranging from the early thirties to the mid‐forties, and often persisting for decades, with a continued dependence on blood transfusions in a proportion of patients.

ALXN1210 is an antibody under development that prevents the destruction of red blood cells (RBCs). The drug is administered by intravenous injection that requires fewer dosing regimen compared to current treatment option. If ALXN1210 is licensed for use in the UK, it could represent an additional treatment option for patients with paroxysmal nocturnal haemoglobinuria.