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Iptacopan
- Development stage
- Approved
- Lead developer
- Novartis
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Iptacopan is an orally administered small-molecule inhibitor of complement factor B, developed for the treatment of complement-mediated diseases. Its primary mechanism is the selective inhibition of factor B in the alternative pathway of the complement system, thereby preventing formation of C3 and C5 convertases. This action reduces both intravascular and extravascular hemolysis in paroxysmal nocturnal hemoglobinuria (PNH) by limiting red blood cell destruction. Iptacopan also reduces proteinuria in primary immunoglobulin A nephropathy (IgAN) and complement 3 glomerulopathy (C3G), conditions characterized by excessive activation of the alternative complement pathway leading to kidney damage. The drug was developed by Novartis and is marketed under the brand name Fabhalta[2][5][7][8].
- Brand names
- Fabhalta
- Other names
- Iptacopan hydrochlorideIptacopan hydrochloride monohydrateXW5CK7C6YHXW-5CK7C6YHXW 5CK7C6YH
02
Targets
CFB (Complement Factor B)
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