Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies

Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies

Abstract: Iptacopan, a first-in-class, oral, selective complement factor B inhibitor, demonstrated efficacy and safety as monotherapy in C5 inhibitor (C5i)–experienced (APPLY-PNH; NCT04558918) and C5i-naive (APPOINT-PNH; NCT04820530) patients with paroxysmal nocturnal hemoglobinuria (PNH). In the AP...

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Main Authors: Antonio M. Risitano, Carlos de Castro, Bing Han, Austin Kulasekararaj, Jaroslaw P. Maciejewski, Phillip Scheinberg, Yasutaka Ueda, Susan Vallow, Georgina Bermann, Marion Dahlke, Rakesh Kumar, Régis Peffault de Latour
Format: Article
Language:English
Published: Elsevier 2025-04-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925000059
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