Iptacopan is a factor B inhibitor used to treat paroxysmal nocturnal hemoglobinuria. Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase.
- Mechanism
- Curator reviewed · 1 reference
Iptacopan binds to Factor B of the alternative complement pathway and regulates the cleavage of C3, the generation of downstream effectors, and the amplification of the terminal pathway. In paroxysmal nocturnal hemoglobinuria, intravascular hemolysis (IVH) is mediated by the downstream membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3b opsonization. Iptacopan acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated EVH and terminal complement-mediated IVH.
- Primary indication
- Iptacopan is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and for the treatment...Curator reviewed · 4 structured indications
- Formula / weight
- C25H30N2O4 · 422.525 g/mol (avg)
- First approval
- Canada, 2025 · United States, 2023 · European Union, 2024
- Also known as
- 4-((2S,4S)-4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl) benzoic acid · BENZOIC ACID, 4-((2S,4S)-4-ETHOXY-1-((5-METHOXY-7-METHYL-1H-INDOL-4-YL)METHYL)-2-PIPERIDINYL)-
- Code names
- LNP-023 · NVP-LNP023 · NVP-LNP023-NX
- Brand names
Resolves to
RENRQMCACQEWFC-UGKGYDQZSA-NSMILESCCO[C@H]1CCN(CC2=C(OC)C=C(C)C3=C2C=CN3)[C@@H](C1)C1=CC=C(C=C1)C(O)=OWhat you can answer from here — as of September 23, 2026
Which drugs share a target with Iptacopan, and which of those have an active Phase 3 trial?
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