Novartis spotlights new PNH data for Fabhalta ahead of EHA

Novartis (NOVN: VX) has unveiled further data for Fabhalta (iptacopan), reinforcing its position in the paroxysmal nocturnal hemoglobinuria (PNH) market and adding momentum to the oral drug’s expanding presence across rare blood and kidney disorders.

The latest readout comes from the Phase IIIb APPULSE-PNH trial, shared ahead of this year’s European Hematology Association congress. The study tracked 52 adults with PNH who switched from injectable anti-C5 therapies - AstraZeneca’s Soliris (eculizumab) or Ultomiris (ravulizumab) - twice-daily oral Fabhalta.

After 24 weeks, over 90% achieved normal or near-normal hemoglobin levels without needing transfusions during the study. Fatigue scores, as measured by a commonly-used metric, improved to levels typically seen in the general population.