Novartis' Fabhalta® Receives FDA Approval as First Oral Monotherapy for Rare Blood Disorder

Novartis has received FDA approval for Fabhalta® (iptacopan), the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta, a Factor B inhibitor, demonstrated superior hemoglobin improvement in the absence of red blood cell transfusions compared to anti-C5 treatments in clinical trials. PNH is a rare blood disorder characterized by hemolysis, bone marrow failure, and thrombosis. The approval offers a new treatment option for PNH patients and addresses the significant unmet need in managing anemia and transfusion dependence. Novartis is also exploring the potential of Fabhalta in other complement-mediated diseases.