(RTTNews) - Novartis (NVS) reported final two-year results from its Phase 3 APPLAUSE IgAN study, showing that Fabhalta (iptacopan) slowed the decline in kidney function by 49.3% compared with placebo. The findings were published in the New England Journal of Medicine and presented as late-breaking data at the 2026 World Congress of Nephrology.
IgA nephropathy is a chronic autoimmune kidney disease in which the immune system deposits IgA proteins in the kidneys, causing inflammation and progressive damage. Over time, this can lead to protein leakage in urine declining kidney function, and ultimately kidney failure. Many patients eventually require dialysis or a transplant, and current supportive treatments do not directly target the underlying disease mechanisms.
Study Overview
The APPLAUSE IgAN is a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating Fabhalta in adults with biopsy-confirmed IgAN who continued to show high levels of proteinuria despite optimized supportive care. Participants were assigned to receive either Fabhalta or placebo and were followed for up to 24 months. The primary measure of effectiveness was the annualized change in estimated glomerular filtration rate (eGFR), a key indicator of kidney function. Secondary measures included the time to kidney failure events and changes in proteinuria.
Fabhalta is an oral Factor B inhibitor designed to block the alternative complement pathway, which plays a central role in the inflammation and tissue damage seen in IgAN.
Two-Year Results
Over the 24-month period, Fabhalta slowed the decline in kidney function by nearly half compared with placebo. Patients receiving Fabhalta experienced an average annual eGFR decline of -3.10 mL/min/1.73 m²/y, while those on placebo declined by -6.12 mL/min/1.73 m²/yr. The treatment also reduced the likelihood of reaching a composite kidney failure endpoint by 43%, with 21.4% of Fabhalta-treated patients experiencing such an event compared with 33.5% in the placebo group.
Proteinuria improvements were also notable. By month nine, 40.7% of patients treated with Fabhalta achieved a 24-hour urine protein-to-creatinine ratio below 1g/g, compared with 23.7% of those receiving placebo. The safety profile remained consistent with earlier findings, with low and comparable rates of adverse events and treatment discontinuation between the two groups.
Expert Commentary
Prof. Vlado Perkovic, Co-Chair of the APPLAUSE IgAN Steering Committee, noted that the results demonstrate Fabhalta's potential to reduce the risk of disease progression and preserve kidney health. Novartis leadership emphasized that the data reflect years of research aimed at developing targeted therapies for IgAN and support the company's broader commitment to advancing treatments for chronic kidney diseases.
Regulatory Status and Next Steps
Fabhalta has already received accelerated approval in the United States and China for reducing proteinuria in adults with IgAN, based on interim results from the APPLAUSE-IgAN trial. Novartis has now submitted the full two-year dataset to the U.S. FDA for traditional approval, and the application has been granted priority review.
Alongside Fabhalta, Novartis continues to advance its multi-asset IgAN portfolio, which also includes Vanrafia (atrasentan), part of its IgAN portfolio, and the investigational compound Zigakibart, which is being developed for IgAN.
NVS has traded between $97.72 and $170.46 over the past year. The stock closed Friday's trading (March 27, 2026) at $148.18, down1.02%. In pre-market trading the stock is at $149.82, up 1.11%.
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