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This review synthesizes clinical trial evidence for Iptacopan, focusing on its transformative role in treating PNH through proximal complement inhibition and its emerging efficacy in IgA nephropathy and C3 glomerulopathy.
This Phase 2 basket trial highlights the divergent efficacy of the oral factor B inhibitor iptacopan, showing significant hemoglobin improvements in Cold Agglutinin Disease but failing to meet platelet response endpoints in Immune Thrombocy
The APPEAR-C3G phase 3 trial demonstrates that oral iptacopan significantly reduces proteinuria and is well tolerated in adults with C3 glomerulopathy, an ultra-rare glomerulonephritis linked to alternative complement pathway overactivation
Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.
