Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH

Core Insights - Novartis announced positive results from the APPULSE-PNH Phase IIIB study, showing that Fabhalta® (iptacopan) significantly improved hemoglobin levels in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched from anti-C5 therapies [1][2][4] Group 1: Study Results - In the APPULSE-PNH study, patients experienced an average hemoglobin improvement of 2.01 g/dL after 24 weeks of treatment with Fabhalta, with 92.7% achieving hemoglobin levels of ≥12 g/dL [1][2][7] - No patients required transfusions during the study, and there were no instances of breakthrough hemolysis or major adverse vascular events [7][9] - Patients reported clinically meaningful improvements in fatigue, with average improvements of 4.88 points at Day 84 and 4.29 points at Day 168, as measured by the FACIT-Fatigue score [2][7] Group 2: Treatment Context - PNH patients have unmet needs that are not fully addressed by existing anti-C5 therapies, which require frequent intravenous infusions and can leave patients anemic [8] - Fabhalta is the first and only oral monotherapy available for PNH treatment, providing a more convenient option for patients compared to traditional therapies [4][8] Group 3: Company Commitment - Novartis is committed to advancing research and innovation to improve the lives of PNH patients, as highlighted by the positive data from the APPULSE-PNH study and other related studies [4][18] - The company emphasizes the importance of Fabhalta in delivering real benefits to patients, reinforcing its efficacy and safety profile [4][14]