Iptacopan from Novartis could become preferred therapy for paroxysmal nocturnal hemoglobinuria

Novartis (NYSE: NVS) recently announced that the pivotal iptacopan Phase 3 APPLY-PNH trial met both its primary and most secondary endpoints in patients with paroxysmal nocturnal hemoglobinuria (PNH). The company recently presented the data at this year’s American Society of Hematology (ASH) meeting.  

Almost all iptacopan recipients had blood-transfusion independence and had clinically meaningful patient-reported-fatigue improvements. There were no serious cases of breakthrough hemolysis (BTH). Iptacopan led to an 80% difference to anti-C5 in the estimated proportion of patients achieving 2 g/dL or more hemoglobin-level increases from baseline. The drug candidate also led to a 67% difference to anti-C5 in the estimated proportion of patients achieving 12 g/dL or more hemoglobin levels. 

“As an oral monotherapy, iptacopan offers PNH patients the convenience of an at-home or anywhere treatment, freeing them from the need to travel to an infusion center for treat…

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