Results showed treatment with iptacopan through 48 weeks caused hemoglobin-level increases, blood transfusion avoidance, and reductions in patient-reported fatigue.
The approval was based on data from the APPLY-PNH trial and APPOINT-PNH study, which showed iptacopan was superior for hemoglobin improvement in the absence of red blood cell transfusions and in transfusion avoidance rate compared to patients who stayed on anti-C5 treatments.