Core Insights - Novartis AG has released results from the APPULSE-PNH Phase 3B study, evaluating the efficacy and safety of Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched from anti-C5 therapies [1][3] Group 1: Study Results - The study showed that after 24 weeks of treatment with Fabhalta, the average hemoglobin (Hb) level improved by 2.01 g/dL, with most patients achieving normal or near-normal levels [3][4] - 92.7% of patients achieved Hb levels ≥12g/dL, and no patients required transfusions during the study [4][5] - Patients reported clinically meaningful improvements in fatigue, reaching levels similar to the general population [4][5] Group 2: Treatment Context - PNH is a rare blood disorder characterized by the destruction of red blood cells (RBCs) due to an acquired mutation in hematopoietic stem cells [2][6] - Current anti-C5 therapies require intravenous infusions every two to eight weeks, which can take approximately four to five hours for treatment visits [6] Group 3: Future Presentations - Data from the APPULSE-PNH study will be presented at the European Hematology Association (EHA) Congress 2025, alongside longer-term data from the APPLY-PNH and APPOINT-PNH Phase 3 studies [5]