Swiss pharmaceutical giant Novartis (NYSE: NVS) has received marketing approval from China for its iptacopan (trade name: Fabhalta) for the treatment of adult paroxysmal nocturnal hemoglobinuria (PNH) in patients previously untreated with complement inhibitor. PNH is a rare and life-threatening blood disorder characterized by anemia, hemoglobinuria, bone marrow failure, and an increased risk of blood clots. The standard treatment for PNH involves anti-complement C5 therapy, yet many patients continue to experience residual anemia, fatigue, and the need for blood transfusions.
Iptacopan is a novel, first-in-class complement factor B (FB) inhibitor that targets the complement pathway’s proximal segment, offering a comprehensive control of both intravascular and extravascular hemolysis, potentially improving upon anti-C5 therapy.
The approval is supported by the pivotal Phase III APPOINT-PNH study, which included a significant patient population from China, accounting for 50% of the subjects. The study demonstrated the efficacy, safety, and tolerability of iptacopan in adult PNH patients who had not previously received complement inhibitor treatment. After 24 weeks of treatment with iptacopan, the majority of patients achieved hemoglobin levels of 12 g/dL or above, and nearly all patients avoided the need for blood transfusions. Additionally, patient-reported fatigue scores improved significantly.- Flcube.com
