Novartis AG has announced positive results from the APPULSE-PNH Phase IIIB study, which evaluated the efficacy and safety of the oral monotherapy Fabhalta® (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH). The study focused on patients with hemoglobin levels of at least 10g/dL who switched from anti-C5 therapies. After 24 weeks of treatment, patients experienced an average hemoglobin level improvement of 2.01 g/dL, with most achieving normal or near-normal levels. Additionally, patients reported improvements in fatigue and maintained control over both intravascular and extravascular hemolysis. No new safety signals were observed. These findings will be presented at the European Hematology Association Congress 2025.
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