Novartis' Fabhalta Delivers Hemoglobin Gains, Reduces Fatigue In Patients With Rare Blood Disorder

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 ste ...