Novartis has announced that the US Food and Drug Administration (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, an oral Factor B inhibitor designed to selectively target the alternative complement pathway, received approval under a priority review designation after an initial FDA accelerated approval in August 2024 for the reduction of proteinuria in primary IgAN1.
“IgAN is a chronic, immune-mediated disease leading to kidney failure that can have a severe impact on patients’ lives,” said Dana Rizk (University of Alabama at Birmingham, Birmingham, USA) a steering committee member in the APPLAUSE-IgAN trial investigating the therapy. “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.”
The approval of Fabhalta was based on data from the Phase III trial, in which results demonstrated statistically significant and clinically meaningful improvement in estimated glomerular filtration rate (eGFR) over two years, with Fabhalta showing an annualised mean change from baseline in eGFR of -3.0 mL/min/1.73 m2/year compared with -5.7 mL/min/1.73 m2/year for placebo. Fabhalta consistently outperformed placebo across key kidney outcomes.
The study also demonstrated a favourable safety profile, consistent with previously reported data. The most common adverse events 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) programme requiring appropriate vaccinations prior to treatment.
“Today’s approval reinforces Fabhalta’s role in preserving kidney function by significantly slowing disease progression, an outcome that matters deeply to patients at risk of long-term kidney damage,” said Victor Bultó, president, US, Novartis. “This milestone underscores the importance of continued innovation for people living with IgAN and our commitment to addressing the underlying drivers of disease.”
