Paroxysmal nocturnal hemoglobinuria (PNH)

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Novartis' Fabhalta Delivers Hemoglobin Gains, Reduces Fatigue In Patients With Rare Blood Disorder
Benzinga· 2025-06-12 18:25
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 ...
Novartis Fabhalta® shows statistically significant and clinically meaningful improvements in hemoglobin in new population of patients with PNH
Globenewswire· 2025-06-12 06:00
It is estimated that approximately 10-20 people per million worldwide live with PNH. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old. In the Phase IIIB APPULSE-PNH study, adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who switched to Fabhalta experienced clinically meaningful improvements in hemoglobin (Hb) levels of 2.01 g/dL on average APPULSE-PNH evaluated Fabhalta in a population with higher baseline hemoglobin (Hb) levels than those enrolled i ...