Class/mechanism: orally bioavailable, highly potent and highly selective factor B inhibitor. Blocks the alternative pathway of complement upstream of both CD55 and CD59. Iptacopan is used in diseases involving abnormal complement-mediated activity, such as paroxysmal nocturnal hemoglobinuria (PNH).
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Diseases for which it is used
Patient drug information
History of changes in FDA indication
- 12/16/2020: FDA breakthrough therapy designation for patients with paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G