Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials
Oral iptacopan provides sustained haemolysis control and improved haemoglobin in PNH patients over 48 weeks, with a favorable safety profile, supporting its role as a new therapeutic option.