Novartis has announced that the US Food and Drug Administration (FDA) has granted traditional approval to Fabhalta (iptacopan) for slowing kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression.
Fabhalta, a first-in-class complement inhibitor, received the approval under the FDA’s Priority Review designation following its accelerated approval in August 2024 for reducing proteinuria (protein in urine) in adults with primary IgAN.
Novartis highlighted that approximately 25 people per million worldwide are newly diagnosed with IgAN, one of the most common autoimmune kidney diseases, each year. Up to 50% of 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 and healthcare systems.
The approval is supported by data from the Phase III APPLAUSE-IgAN study, which demonstrated statistically significant and clinically meaningful preservation of kidney function over two years. Fabhalta achieved an annualized mean decline in estimated glomerular filtration rate (eGFR) of -3.0 mL/min/1.73 m²/year, compared with -5.7 mL/min/1.73 m²/year for placebo. The treatment consistently outperformed placebo across key kidney outcomes.
Fabhalta slowed eGFR decline by 48% compared with placebo over two years, demonstrating its ability to preserve kidney function. Clinically meaningful reductions in proteinuria were observed as early as two weeks after treatment initiation and were sustained throughout the treatment period.
“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.
How Fabhaltaworks
Fabhalta (iptacopan) is an oral Factor B inhibitor designed to selectively target the alternative complement pathway, one of key drivers of glomerular inflammation and kidney damage in IgAN. By inhibiting Factor B, Fabhalta is intended to reduce ongoing complement-mediated injury and slow disease progression.
Fabhalta has received regulatory approvals in multiple complement-mediated diseases, including IgAN, and is currently being evaluated across a range of rare kidney disorders.
Alongside Fabhalta, Novartis is expanding its IgAN portfolio with Vanrafia (atrasentan) and the investigational therapy zigakibart. The company also offers patient support programmes aimed at improving access to Fabhalta, with nearly 100% of eligible US patients paying $10 or less per month for the treatment.
