Novartis AG ( NVS ) on Thursday released results from APPULSE-PNH, a Phase 3B study evaluating the efficacy and safety of twice-daily oral monotherapy Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) with Hb levels ≥10g/dL who switched from anti-C5 therapies (eculizumab or ravulizumab).

The company released topline data in December 2024.

What Happened: PNH is a rare complement-mediated blood disorder. People with PNH have an acquired mutation in some of their hematopoietic stem cells that causes them to produce RBCs susceptible to premature destruction.

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This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver).

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.

No patients required transfusion during the study, and the vast majority (92.7%) achieved Hb ≥12g/dL, reaching normal or near-normal levels. Patients treated with Fabhalta also reported clinically meaningful improvements in fatigue (as measured by FACIT-Fatigue score) through Day 168, reaching absolute levels similar to those reported in the general population.

Furthermore, patients treated with Fabhalta maintained intravascular hemolysis control and resolved extravascular hemolysis control, as demonstrated by lactate dehydrogenase levels (