After switching to Iptacopan: Hemoglobin level improved clinically relevant

Special report | With kind support from: Novartis Pharma GmbH, Nuremberg

Paroxysmal nocturnal hemoglobinuria (PNH) After switching to Iptacopan: Hemoglobin level improved clinically relevant

Since May 2024, iptacopan has been the first oral factor B inhibitor approved as monotherapy for complement inhibitor-naive or previously treated patients with PNH who have hemolytic anemia. Data from the APPULSE-PNH study, recently presented at the 2025 EHA (European Hematology Association) Congress, show that even previously treated patients with hemoglobin (Hb) levels ≥10 g/dL benefit from switching to iptacopan. Switching to the factor B inhibitor enables a clinically relevant increase in Hb levels, even in patients who responded well to previous therapy, with associated improvements in quality of life and reduced fatigue.