Novartis recently shared promising findings from the Phase III APPLAUSE-IgAN study of Fabhalta® (iptacopan), a Factor B inhibitor targeting the alternative complement pathway, in patients with IgA nephropathy (IgAN). In this interim analysis, patients treated with Fabhalta experienced a significant 38.3% reduction in proteinuria (measured by 24-hour urine protein to creatinine ratio [UPCR]) after 9 months compared to those receiving placebo alongside supportive care.
Reducing proteinuria is crucial in IgAN management as it correlates with the risk of kidney failure. Fabhalta demonstrated a favorable safety profile, consistent with previous data, making it a promising treatment option. These findings were presented during a session at the World Congress of Nephrology in Buenos Aires, Argentina.
Professor Dana Rizk, an Investigator and APPLAUSE-IgAN Steering Committee Member, highlighted the significance of Fabhalta’s targeted approach to the alternative complement pathway in IgAN treatment.
The interim analysis involved 250 patients for efficacy and 443 for safety assessment. The study continues in a double-blind manner, with limited interim results available. Submission for accelerated approval to the FDA is ongoing, with the primary endpoint, evaluating Fabhalta’s ability to slow IgAN progression, expected upon study completion in 2025.
David Soergel, M.D., Global Head of Novartis’ Cardiovascular, Renal, and Metabolism Development Unit, emphasized the importance of offering diverse therapies to address patients’ evolving needs throughout the disease course.
Additionally, Novartis presented real-world data on IgAN and C3 glomerulopathy (C3G) studies at the congress and plans to share further insights from its renal portfolio in upcoming medical meetings.
The APPLAUSE-IgAN study (NCT04578834) is a Phase III trial evaluating Fabhalta’s efficacy and safety in 518 adult primary IgAN patients. Fabhalta, an oral Factor B inhibitor, is also being investigated for other rare diseases including C3G, atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN).
IgAN, a rare kidney disease, affects approximately 25 people per million annually worldwide, with up to 30% progressing to kidney failure within a decade. Novartis is committed to addressing unmet needs in renal care through its innovative portfolio, including Fabhalta and other investigational therapies such as atrasentan and zigakibart, currently in Phase III development. The company aims to provide effective treatments to improve outcomes and extend the lives of patients with kidney diseases.
