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The proximal complement inhibitor, IPTACOPAN is the pioneer in treating a vast variety of complement mediated diseases 1,13. The fact that it acts upstream in the complement synthesis pathway by inhibiting Factor B 1, prevents the formation of C3 convertase which plays a crucial role in the pathogenesis of Paroxysmal Nocturnal Hemoglobinuria (PNH), IgA nephropathy, Membranous Nephropathy, and Immune thrombocytopenic purpura 5,6,9,14. Studies have shown that the usage of Iptacopan for PNH has led to an increase in the Hemoglobin (Hb) levels by at least a minimum of 2gm/dl and majority of the patients in the treatment group did not have to receive transfusion 2. It is effective in preventing both extravascular and intravascular hemolysis 14. A phase 2 study on Iptacopan for treatment of IgA nephropathy showed a statistically signicant dose-response effect with 10mg, 20mg, 100mg and 200mg in reducing urine protein creatinine ratio (UPCR) 3. Both in Membranous nephropathy and Immune thrombocytopenic purpura patients who are refractory to current standard treatments, Iptacopan has proved to be efcient in improving the clinical status of such patients 6,9. In this review below we discuss the potential ways in which Iptacopan has proved to be effective in treating some of the complement mediated diseases.
Varshitha et al. (Mon,) studied this question.