29 Dec 2025
byAudrey Abella
In a subgroup analysis of patients with paroxysmal nocturnal haemoglobinuria (PNH) with a history of aplastic anaemia and concomitant immunosuppressive therapy (AA + IST), long-term treatment with iptacopan yields clinically meaningful improvements in haemoglobin levels, transfusion needs, and haemolysis control, with safety outcomes comparable to those in individuals without AA.