Research molecule of Novartis, Iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naive paroxysmal nocturnal hemoglobinuria patients, as per recent trials.
The trial met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints. These data were presented at the 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT).
With iptacopan treatment, an estimated 92.2% of patients (95% confidence interval) achieved a 2 g/dL or more hemoglobin-level increase from baseline without the need for red blood cell transfusions after the 24-week core treatment period.
PNH (paroxysmal nocturnal hemoglobinuria) is a rare, chronic, and serious complement-mediated blood disorder. It has a significant unmet need despite treatment with anti-C5s, as a large proportion of people with PNH remain anemic, fatigued, and dependent on blood transfusions. It is estimated that approximately 10-20 people per million worldwide live with PNH.
Iptacopan demonstrated a tolerability and safety profile consistent with previously reported data. The most commonly reported adverse events (AEs) were infections (in 40.0% of patients, mainly COVID-19 [15.0%] and upper respiratory tract infection [12.5%]), headache (27.5%) and diarrhea (7.5%), with four serious AEs reported. No patients discontinued iptacopan in the 24-week treatment period.
Discovered at the Novartis Institutes for BioMedical Research, iptacopan is currently in development for a number of other complement-mediated diseases (CMDs) where significant unmet needs exist, including kidney diseases C3 glomerulopathy (C3G), IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), lupus nephritis (LN), and blood disorders immune thrombocytopenic purpura (ITP) and cold agglutinin disease (CAD).
