Iptacopan (Fabhalta)
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)
Toxicity management
Diseases for which it is established
History of changes in FDA indication
- 2023-12-05: Approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). (Based on APPLY-PNH & APPOINT-PNH)
Also known as
- Code name: LNP-023
- Brand name: Fabhalta