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Data at EBMT show primary endpoint met – estimated* 92.2% of complement-inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) achieving 2...

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

Trial met both primary and most secondary endpoints, showing iptacopan provided transfusion-free hemoglobin-level increases in vast majority of adult paroxysmal nocturnal hemoglobinuria (PNH) patients

Phase III APPOINT-PNH study of investigational oral monotherapy iptacopan met its primary endpoint; second positive Phase III topline readout for iptacopan in...

Posted December 16th, 2020 for Novartis Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disorder, resulting in debilitating symptoms that can impact patients’ quality of life 1–3 C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need 4-5 With potential to be the first oral treatment for a range of complement-driven diseases, complement factor B inhibitor iptacopan targets the underlying...