Pharmacology
Factor B inhibitor; iptacopan binds to Factor B of the alternative complement pathway, regulating C3 cleavage and inhibiting downstream effectors to control both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis in PNH, and to inhibit complement-driven glomerular injury in IgAN and C3G.
Complement Factor D inhibitor; danicopan binds reversibly to Factor D and selectively inhibits the alternative complement pathway, preventing C3 convertase formation, reducing C3 fragment opsonization, and thereby controlling extravascular hemolysis in PNH when used as add-on therapy to a terminal complement inhibitor.