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
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