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, demonstrating the efficacy and safety of Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched from anti-C5 therapies [1][4][9] - The study showed an average improvement in hemoglobin (Hb) levels of 2.01 g/dL after 24 weeks of treatment, with 92.7% of patients achieving Hb levels ≥12 g/dL [1][2][7] - Fabhalta is the first and only oral monotherapy available for PNH treatment, providing a significant alternative to existing intravenous therapies [4][8][14] Group 1: Study Results - The APPULSE-PNH study involved 52 participants who received Fabhalta for 24 weeks, with no patients requiring transfusions or experiencing breakthrough hemolysis [9][10] - Patients reported clinically meaningful improvements in fatigue, with average FACIT-Fatigue score improvements of 4.88 points at Day 84 and 4.29 points at Day 168 [2][7] - The treatment maintained intravascular hemolysis control and resolved extravascular hemolysis, as indicated by lactate dehydrogenase levels and a reduction in absolute reticulocyte count [3] Group 2: Clinical Context - PNH is a rare, chronic blood disorder affecting approximately 10-20 people per million worldwide, often diagnosed in individuals aged 30-40 [5][6] - Current anti-C5 therapies, such as eculizumab and ravulizumab, do not fully address the needs of all PNH patients, leaving many anemic and dependent on blood transfusions [8] - The introduction of Fabhalta addresses these unmet needs by offering an oral treatment option that can improve hemoglobin levels without the need for transfusions [2][4][8] Group 3: Company Commitment - Novartis is committed to advancing research and innovation to improve the lives of patients with PNH, as highlighted by the positive data from the APPULSE-PNH study [4][18] - The company plans to present longer-term data from related Phase III studies at the European Hematology Association Congress 2025, reinforcing the efficacy and safety profile of Fabhalta [4][1] - Fabhalta has received regulatory approvals in the US and Europe for the treatment of adults with PNH, further establishing Novartis's position in the rare disease market [14][18]