Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Haemoglobinuria: 48-Week Outcomes from APPLY-PNH and APPOINT-PNH Phase 3 Trials

Highlights

• Iptacopan, an oral factor B inhibitor, demonstrated durable control of haemolysis and improved haemoglobin levels over 48 weeks in both anti-C5-experienced and complement-inhibitor-naive PNH patients.
• High proportions of patients achieved clinically meaningful haemoglobin increases (≥2 g/dL) and normalization (≥12 g/dL) without new safety concerns.
• Breakthrough haemolysis and severe adverse events were infrequent, with no treatment discontinuations due to adverse events or deaths.

Study Background and Disease Burden