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Iptacopan sustains hemolysis control in PNH out to six years

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Two long-term follow-up studies presented at EHA 2026 show that iptacopan durably controls hemolysis in paroxysmal nocturnal hemoglobinuria (PNH), with stable hematologic responses maintained over follow-up of up to four and six years. In the six-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 hemoglobin at or above 12 g/dL, LDH below 1.5 times the upper limit of normal, and normalized absolute reticulocyte counts, while 88.5% were free of transfusion dependence. FACIT-F fatigue scores improved significantly from month 6 and remained stable thereafter. The data support iptacopan's durability in PNH; further detail beyond these follow-up results has not been reported.

Written by a model from the reports · updated 4 hours ago

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Oct 24
  1. mp.weixin.qq.comPick
    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.

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