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Iptacopan improves hematologic and clinical outcomes in paroxysmal nocturnal hemoglobinuria

1. In two clinical trials, iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH) improved hemoglobin levels compared to anti-C5-treated patients and those without complement inhibitors. 2. Patients who received iptacopan generally exhibited improved secondary outcomes, including less fatigue, reduced reticulocyte and bilirubin levels, and decreased breakthrough hemolysis. Evidence Rating Level: 1 (Excellent) Study Rundown: PNH
Minute Medicine Inc Rating Level Complement Inhibitors Internal Medicine Paroxysmal Nocturnal Hemoglobinuria

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Novartis Pharma AG: Novartis Phase III APPOINT-PNH trial shows investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naïve PNH patients

Data at EBMT show primary endpoint met - estimated* 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2 g/dL or more hemoglobin-level increase from baseline
South Korea France General United States Satoshi Sugimoto Julie Masow Giuseppe Moscati

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