About Iptacopan API
Therapeutic CategoryHaematology/Nephrology

CAS Number
1644670‑37‑0
API Technology
Small‑molecule synthetic API
Dose Form
口服固体/胶囊
Dr Reddy's Development Status
开发中
Mechanism of action
Iptacopan binds to complement factor B (FB), a key component of the alternative complement pathway.
This inhibits formation of the C3 convertase (C3bBb), thereby:
Reducing C3 cleavage and C3b generation
Preventing downstream formation of C5 convertase and the membrane attack complex (MAC)
By acting upstream (proximal) in the complement cascade, iptacopan controls:
C3b‑mediated extravascular hemolysis
Terminal complement‑mediated intravascular hemolysis
Importantly, it selectively blocks the alternative pathway, leaving the classical and lectin pathways intact to preserve host immune defense.
Indication
Approved indications (FDA / EMA):
Paroxysmal nocturnal hemoglobinuria (PNH) in adults
Reduction of proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression
Complement 3 glomerulopathy (C3G) to reduce proteinuria
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