The U.S. FDA has approved Novartis’ oral Fabhalta (iptacopan) for the treatment of adults with C3 glomerulopathy (C3G), rare kidney disease, marking the first and only treatment approved for the condition.
C3G is a progressive and ultra-rare kidney disease that, until now, has had no approved treatments. Prior to Fabhalta, patients had to rely on supportive care, broad immunosuppression, and symptom management. Fabhalta is the only oral inhibitor of the alternative complement pathway to selectively target what is thought to be the underlying cause of the disease.
The approval was supported by the pivotal phase 3APPEAR-C3G study, which evaluated the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G1. Treatment with Fabhalta resulted in clinically meaningful proteinuria (protein in urine) reduction, which was seen as early as 14 days and sustained at 12 months. Similarly, in the open-label period, proteinuria reduction was seen in participants who switched to Fabhalta.
This is the third FDA approval for Fabhalta. The complement factor B inhibitor was first approved in 2023, as a treatment for paroxysmal nocturnal hemoglobinuria, a rare blood disorder caused by a mutation in the PIG-A gene. In August 2024, the drug won a second approval for use in primary immunoglobulin A nephropathy (IgAN), a chronic kidney disease that can lead to kidney failure.
Fabhalta also received a positive CHMP opinion in C3G by the European Medicines Agency last month.