FDA grants traditional approval for Fabhalta (iptacopan) as first and only complement inhibitor to significantly slow kidney function decline in primary IgAN – Novartis
Novartis announced that the FDA has granted traditional approval for Fabhalta (iptacopan) to slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. Fabhalta, a first-in-class complement inhibitor, received approval under a priority review designation after an initial FDA accelerated approval in August 2024 for the reduction of proteinuria in primary IgAN.
“IgAN is a chronic, immune-mediated disease leading to kidney failure that can have a severe impact on patients’ lives,” said Dana Rizk, M.D., Professor of Medicine in the Division of Nephrology at the University of Alabama at Birmingham and APPLAUSE-IgAN Steering Committee Member. “The ability to significantly slow kidney function decline is a critical treatment goal. This approval of Fabhalta reinforces the importance of targeting underlying disease mechanisms, including complement activation, in treating IgAN to help preserve kidney health.”
Each year, approximately 25 people per million worldwide are newly diagnosed with IgAN, one of the most common autoimmune kidney diseases. Up to 50 percent of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring dialysis and/or kidney transplantation, placing a significant burden on patients.
“This milestone is a moment of great hope for the IgAN community,” said Bonnie Schneider, Director and Co-Founder, IgA Nephropathy Foundation. “For patients and families impacted by this progressive disease, knowing that Fabhalta can help preserve kidney function brings renewed confidence and optimism for the future of the IgAN treatment landscape.”
Data supporting approval
The approval of Fabhalta was based on data from the Phase III APPLAUSE-IgAN study. Results demonstrated statistically significant and clinically meaningful improvement in estimated glomerular filtration rate (eGFR) over two years, with Fabhalta showing an annualized mean change from baseline in eGFR of -3.0 mL/min/1.73 m2/yr compared with -5.7 mL/min/1.73 m2/yr for placebo. Fabhalta consistently outperformed placebo across key kidney outcomes.
The APPLAUSE-IgAN study showed that Fabhalta has a favorable safety profile, consistent with previously reported data. The most common adverse events with Fabhalta in patients with IgAN were abdominal pain, dizziness and nausea. Fabhalta may increase the risk of serious infections caused by encapsulated bacteria and is available only through a Risk Evaluation and Mitigation Strategy (REMS) program requiring appropriate vaccinations prior to treatment.
