Results showed treatment with iptacopan through 48 weeks caused hemoglobin-level increases, blood transfusion avoidance, and reductions in patient-reported fatigue.
Iptacopan elicited improved hemoglobin levels measuring at least 2 g/dL higher vs baseline, leading to transfusion independence after 24 weeks in approximately 92.2% of patients with complement inhibitor–naïve paroxysmal nocturnal hemoglobinuria.
The APPOINT-PNH study showed that twice-daily LNP023 (Iptacopan) helped patients with paroxysmal nocturnal hemoglobinuria control intravascular hemolysis.
Data at EBMT show primary endpoint met – estimated 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2.
Data at EBMT show primary endpoint met - estimated 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2 g/dL or more hemoglobin-level increase from baseline