Novartis’ Fabhalta Approved by FDA for C3 Glomerulopathy Treatment

Swiss pharmaceutical giant Novartis’ (NYSE: NVS) iptacopan, marketed as Fabhalta, has received approval from the US Food and Drug Administration (FDA) for the treatment of adults with C3 glomerulopathy (C3G). This marks the first and only approved therapy specifically targeting this rare kidney disease, which is characterized by a poor prognosis and often leads to kidney failure within a decade of diagnosis.

Drug Mechanism and Clinical Significance
Fabhalta is an oral inhibitor of the alternative complement pathway, designed to selectively address the underlying cause of C3G. The FDA’s approval is based on results from the pivotal Phase III APPEAR-C3G study, which demonstrated clinically meaningful reductions in proteinuria as early as 14 days into treatment, with sustained benefits at 12 months. The drug also exhibited a favorable safety profile with no new safety signals.-Fineline Info & Tech