Novartis Fabhalta® (iptacopan) meets Phase III primary endpoint, slows kidney function decline in patients with IgA nephropathy (IgAN)

In APPLAUSE-IgAN final analysis, Fabhalta demonstrated statistically significant, clinically meaningful improvement in estimated glomerular filtration rate (eGFR) slope vs. placebo over two years1eGFR is key marker of kidney function; IgAN is progressive autoimmune kidney disease that leads to kidney failure in many patients1-3Fabhalta is first and only approved complement inhibitor for adults with IgAN and has potential to delay disease progression4,5Fabhalta received accelerated approval for reduction of proteinuria in adults with IgAN in US in 2024; data support 2026 submission for traditional FDA approval4,5

Basel, October 16, 2025 – Novartis today announced positive final results from APPLAUSE-IgAN, a Phase III study evaluating Fabhalta® (iptacopan) in adults living with IgA nephropathy (IgAN). Fabhalta, an oral alternative complement pathway inhibitor, demonstrated statistically significant, clinically meaningful superiority compared to placebo in slowing IgAN progression measured by annualized total slope of estimated glomerular filtration rate (eGFR) decline over two years1.

“Progressive diseases such as IgAN present an urgent need for interventions that can ultimately improve kidney health. Many people with IgAN commonly experience fear and anxiety of disease progression,” said Ruchira Glaser, Development Unit Head, Cardiovascular, Renal & Metabolic, Novartis. “We are excited about today’s positive Phase III APPLAUSE-IgAN results showing slowed eGFR decline, which add to the growing evidence of Fabhalta as a targeted therapy to preserve long-term kidney function, giving hope to people living with this condition.” 

Novartis intends to use these data to support Fabhalta submissions in 2026. Alongside Fabhalta, Novartis continues to advance its multi-asset IgAN portfolio, which also includes Vanrafia® (atrasentan) and investigational compound zigakibart.

IgAN is a progressive autoimmune kidney disease with approximately 25 per million people newly diagnosed worldwide each year3. IgAN is highly debilitating as it leads to glomerular inflammation, proteinuria, and a gradual decline in eGFR2. Up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring dialysis or kidney transplantation as part of long-term disease management2,6,7. Furthermore, people living with IgAN often face mental, social, and economic challenges2,8. Supportive care does not address the underlying causes of the disease and often fails to slow disease progression, reinforcing the need for more targeted therapies for IgAN3,9.

In APPLAUSE-IgAN, Fabhalta was well tolerated with a favorable safety profile in line with previously reported data10. Full data from the APPLAUSE-IgAN final analysis will be presented at future medical meetings.

About Fabhalta® (iptacopan)
Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway10.

Discovered at Novartis, Fabhalta received US Food and Drug Administration (FDA) and European Commission (EC) approval in December 2023 and May 2024, respectively, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta also received accelerated approval in the US in August 2024, and in China in September 2025, for the reduction of proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression (generally UPCR ≥1.5 g/g4,5,11,12. In 2025, Fabhalta received FDA and EC approval as well as approvals in China and Japan for the treatment of adults with C3 glomerulopathy (C3G), making it the first treatment approved for this condition13-15.

Fabhalta is being studied in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN)16-18. Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions16-18.

About APPLAUSE-IgAN
APPLAUSE-IgAN (NCT04578834) is a Phase III multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of twice-daily oral Fabhalta (200 mg) in 477 adult primary IgAN patients (main study population). Patients were randomized to Fabhalta or placebo, on top of supportive care (a stable dose of maximally-tolerated renin-angiotensin system (RAS) inhibitor therapy with or without a stable dose of SGLT2i)1.

The two primary endpoints of the study for the interim and final analysis, respectively, are proteinuria reduction at 9 months as measured by UPCR, and the annualized total eGFR slope over 24 months10,18. During the final analysis, the following secondary endpoints were assessed: proportion of participants reaching UPCR