European Medicines Agency's CHMP recommends marketing authorization for Novartis' Fabhalta® (iptacopan) to treat PNH adults with hemolytic anemia.

European Medicines Agency's CHMP recommends granting a marketing authorization for Novartis' Fabhalta® (iptacopan) for treating adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia. This oral monotherapy, based on Phase III APPLY-PNH and APPOINT-PNH studies, may normalize hemoglobin levels and reduce transfusion needs in PNH patients. Fabhalta could be a practice-changing treatment for the rare and debilitating chronic blood disorder, which is currently treated with anti-C5 therapies.