Iptacopan (LNP023)

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General information

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).
Route: oral

For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias such as Micromedex, Lexicomp, Medscape, UpToDate (courtesy of Lexicomp)

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