EHA 2026: iptacopan long-term follow-up in PNH shows sustained hemolysis control

Two long-term follow-up studies presented at EHA 2026 show that iptacopan durably controls hemolysis in PNH, maintaining stable hematologic responses over follow-up of up to 4 and 6 years. In the 6-year follow-up of the phase 2 and extension studies, median treatment duration was 66.1 months; at year 6, 85.7% of patients had Hb at or above 12 g/dL, LDH below 1.5 x ULN and normalized ARC, 88.5% were free of transfusion dependence, and FACIT-F scores improved significantly from month 6 and remained stable.