Régis Peffault de Latour, Alexander Röth, Austin G Kulasekararaj, Bing Han, Phillip Scheinberg, 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, Flore Sicre de Fontbrune, Luana Marano, Ferras Alashkar, Shreyans Gandhi, Roochi Trikha, Chen Yang, Hui Liu, Richard J Kelly, Britta Höchsmann, Cécile Kerloeguen, Partha Banerjee, Rafael Levitch, Rakesh Kumar, Zhixin Wang, Christine Thorburn, Samopriyo Maitra, Shujie Li, Aurelie Verles, Marion Dahlke, Antonio M Risitano
BACKGROUND: Persistent hemolytic anemia and a lack of oral treatments are challenges for patients with paroxysmal nocturnal hemoglobinuria who have received anti-C5 therapy or have not received complement inhibitors. Iptacopan, a first-in-class oral factor B inhibitor, has been shown to improve hemoglobin levels in these patients. METHODS: In two phase 3 trials, we assessed iptacopan monotherapy over a 24-week period in patients with hemoglobin levels of less than 10 g per deciliter...
March 14, 2024: New England Journal of Medicine