Bone Marrow Failure Disorders

In this multicenter, open-label, single-arm, phase 2 trial at centers in Italy, France, and Germany, researchers enrolled adult patients 18-80 years of age with paroxysmal nocturnal hemoglobinuria (PNH) who showed signs of active hemolysis despite receiving treatment with eculizumab. During the 13-week study period (between May 2018 and April 2019) a total of 10 patients received iptacopan monotherapy at 200 mg twice daily. Iptacopan resulted in marked reduction of LDH from baseline (indicating reduction of chronic residual intravascular hemolysis), associated with significant improvement of hemoglobin concentrations. All biomarkers of hemolysis improved on treatment with iptacopan.