Novartis announces results from APPULSE-PNH study of Fabhalta

Novartis (NVS) announced positive results from APPULSE-PNH, a Phase IIIB study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta in adult patients with paroxysmal nocturnal hemoglobinuria with Hb levels greater than or equal to10g/dL who switched from anti-C5 therapies. After 24 weeks of treatment with Fabhalta, the Hb level improved on average by 2.01 g/dL with most patients achieving normal or near-normal levels. Data will be presented at the European Hematology Association, EHA, Congress 2025. In the Phase IIIB APPULSE-PNH study, adult patients with paroxysmal nocturnal hemoglobinuria who switched to Fabhalta experienced clinically meaningful improvements in hemoglobin levels of 2.01 g/dL on average. APPULSE-PNH evaluated Fabhalta in a population with higher baseline hemoglobin levels than those enrolled in the pivotal Phase III program, expanding the clinical evidence base. No patients treated with Fabhalta required transfusions, experienced breakthrough hemolysis or had any major adverse vascular events during the treatment period. Patients treated with Fabhalta reported an average improvement in fatigue from baseline of 4.88 points at Day 84 and 4.29 points at Day 168 of the study.