Novartis investigational iptacopan provides clinically meaningful increases in hemoglobin levels in complement-inhibitor-naïve patients with PNH

Novartis' Phase III APPOINT-PNH study showed iptacopan, an oral monotherapy, significantly increased hemoglobin levels in complement-inhibitor-naïve PNH patients without blood transfusions. Iptacopan also demonstrated superiority over anti-C5 therapies in the APPLY-PNH study, offering potential as the first oral monotherapy for PNH.