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Trial data released for potentially "practice-changing" PNH medicine - Vimarsana News

Trial data released for potentially "practice-changing" PNH medicine

Iptacopan achieved "improvement of anaemia never seen before with anti-C5s” in PNH, showed Novartis data presented at 2023 EBMT.

Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Paroxysmal Nocturnal Hemoglobinuria - Vimarsana News

Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Paroxysmal Nocturnal Hemoglobinuria

Iptacopan elicited improved hemoglobin levels measuring at least 2 g/dL higher vs baseline, leading to transfusion independence after 24 weeks in approximately 92.2% of patients with complement inhibitor–naïve paroxysmal nocturnal hemoglobinuria.