Novartis’ Fabhalta (iptacopan) has secured traditional approval from the US FDA as the first and only complement inhibitor shown to significantly slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression.
The approval marks a major milestone for patients living with IgAN, a chronic autoimmune kidney disease that can progress to kidney failure. Fabhalta demonstrated a 48% reduction in estimated decline of kidney function compared with placebo over two years, highlighting its potential to preserve kidney health in a disease where treatment options remain limited.
The FDA decision follows Fabhalta’s accelerated approval in August 2024 for reducing proteinuria — excess protein in urine — in primary IgAN. The latest approval expands its role by confirming its ability to slow disease progression.
'IgAN is a chronic, immune-mediated disease leading to kidney failure that can have a severe impact on patients’ lives,” said Dana Rizk, 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.”
The approval was supported by results from the Phase III APPLAUSE-IgAN study, which showed statistically significant and clinically meaningful improvements in estimated glomerular filtration rate (eGFR), a key measure of kidney function.
Over two years, patients receiving Fabhalta experienced an annualized mean eGFR change from baseline of -3.0 mL/min/1.73 m²/year, compared with -5.7 mL/min/1.73 m²/year for those receiving placebo.
The study also showed rapid reductions in proteinuria, with clinically meaningful improvements observed as early as two weeks after starting treatment and sustained throughout the treatment period.
Fabhalta works by selectively blocking Factor B in the alternative complement pathway — a part of the immune system believed to play a key role in the inflammation and kidney damage associated with IgAN.
IgAN affects approximately 25 people per million worldwide each year and is among the most common autoimmune kidney diseases. For patients with persistent proteinuria, up to 50% may progress to kidney failure within 10 to 20 years of diagnosis, potentially requiring dialysis or kidney transplantation.
“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.”
The approval reinforces Novartis’ growing focus on kidney disease therapies designed to address the biological causes of disease rather than only managing late-stage complications.
“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.”
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