Novartis Receives FDA Approval for Fabhalta in Paroxysmal Nocturnal Hemoglobinuria

Novartis today announced that the FDA has approved Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). The approval was based on results from the APPLY-PNH and APPOINT-PNH trials, which demonstrated that iptacopan significantly increased hemoglobin levels and reduced transfusion requirements compared to anti-C5 therapy.