english.prescrire.org > Spotlight > 100 most recent > Iptacopan (Fabhalta°) in paroxysmal nocturnal haemoglobinuria

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Iptacopan (Fabhalta°) in paroxysmal nocturnal haemoglobinuria

 Marketing Authorisations  Paroxysmal nocturnal haemoglobinuria is a rare, potentially fatal condition in which patients produce red blood cells that are unusually susceptible to haemolysis. Treatment is mainly based on blood transfusions and intravenous eculizumab or ravulizumab. Iptacopan has been authorised in the European Union as an oral treatment for adults with paroxysmal nocturnal haemoglobinuria with haemolytic anaemia. 
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 ©Prescrire 1 December 2025

Source: "Iptacopan (Fabhalta°) in paroxysmal nocturnal haemoglobinuria" Prescrire Int 2025; 34 (276): 289-290. Subscribers only.

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