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Alternate Names: Fabhalta, Iptacopanum
CAS No: 1644670-37-0
PubChem CID: 90467622
Mol Formula: C25H30N2O4
Mol Weight: 422.5 g/mol
IUPAC Name: 4-[(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl]benzoic acid
Commercial / Development Status: Commercial APIs
Source / Manufacturing Origin: Synthetic APIs
API Description: Iptacopan is a first-in-class, oral small-molecule factor B inhibitor that targets the alternative complement pathway. It binds to complement factor B, preventing the formation of C3 convertase, which stops C3b-mediated extravascular hemolysis and downstream C5-mediated intravascular hemolysis, effectively treating diseases like paroxysmal nocturnal hemoglobinuria (PNH).
