Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors

Authors: Antonio M Risitano, Austin G Kulasekararaj, Phillip Scheinberg, Alexander Röth, Bing Han, Jaroslaw P Maciejewski, Yasutaka Ueda, Carlos M de Castro, Eros Di Bona, Rong Fu, Li Zhang, Morag Griffin, Saskia M C Langemeijer, Jens Panse, Hubert Schrezenmeier, Wilma Barcellini, Vitor A Q Mauad, Philippe Schafhausen, Suzanne Tavitian, Eloise Beggiato, Lee Ping Chew, Anna Gaya, Wei-Han Huang, Jun Ho Jang, Toshio Kitawaki, Abdullah Kutlar, Rosario Notaro, Vinod Pullarkat, Jörg Schubert, Louis Terriou, Michihiro Uchiyama, Lily Wong Lee Lee, Eng-Soo Yap, Camilla Frieri, Luana Marano, Flore Sicre de Fontbrune, Shreyans Gandhi, Roochi Trikha, Ferras Alashkar, Chen Yang, Hui Liu, Richard J Kelly, Britta Höchsmann, Tomasz Lawniczek, Navin Mahajan, Susan Solar-Yohay, Cécile Kerloëguen, Philippe Ferber, Rakesh Kumar, Zhixin Wang, Christine Thorburn, Samopriyo Maitra, Shujie Li, Aurelie Verles, Marion Dahlke, Régis Peffault de Latour

Published: 2025-05-28

DOI: 10.1016/s2352-3026(25)00081-x

Source: Full article

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Abstract

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