Novartis’ (NYSE: NVS) Fabhalta (iptacopan) has received approval from China’s National Medical Products Administration (NMPA) for the treatment of adults with C3 glomerulopathy (C3G). This approval follows its previous nod for paroxysmal nocturnal hemoglobinuria (PNH) in April last year, further expanding the drug’s therapeutic applications.
Disease Background
C3G is a progressive and ultra-rare kidney disease with a poor prognosis. Approximately half of patients progress to kidney failure within 10 years of diagnosis, requiring lifelong dialysis or kidney transplantation. Even with transplantation, recurrence rates remain high at 50% to 70%.
Mechanism of Action
Fabhalta, an oral specific complement factor B inhibitor, directly targets the overactivation of the alternative pathway (AP) in the immune mechanism, a key driver of C3G. By suppressing downstream reactions and product formation, it helps prevent inflammation and kidney damage.-Fineline Info & Tech