Novartis has achieved a major milestone in the treatment of IgA nephropathy (IgAN), reporting a 38.3% reduction in proteinuria in a pivotal Phase III trial. The study, dubbed APPLAUSE-IgAN, tested Fabhalta (iptacopan), an investigational Factor B inhibitor, demonstrating not only effectiveness in reducing protein levels in urine—a key marker for kidney disease progression—but also a favorable safety profile. These findings were shared during a late-breaking clinical trials session at the World Congress of Nephrology in Buenos Aires, Argentina.

Proteinuria, the presence of excess proteins in urine, is an important indicator of kidney health. Its reduction is increasingly seen as vital for slowing the progression towards kidney failure, a significant risk in IgAN patients. Novartis’s Fabhalta has shown potential in this area, which could support its candidacy for accelerated approval by the FDA.

The interim analysis of the APPLAUSE-IgAN study included data from 250 patients evaluated for efficacy and 443 for safety. The primary goal of Fabhalta treatment is to slow the decline in kidney function, measured by the annualized total estimated glomerular filtration rate (eGFR) slope over 24 months. This study, still ongoing in a double-blind manner, is set to complete in 2025, with hopes for early FDA approval.

Professor Dana Rizk, a key investigator in the study, highlighted the debilitating nature of IgAN and its treatments. According to Professor Rizk, “The loss of kidney function, together with potential side effects of IgAN treatments available until recently, significantly impact patients’ lives.” Dr. David Soergel, Global Head of the Cardiovascular, Renal and Metabolism Development Unit at Novartis, also emphasized the need for varied treatments as IgAN progresses over years, with patient needs evolving over time.

Novartis is not limiting its focus to Fabhalta alone; the company’s renal portfolio includes a variety of other potential treatments aimed at different stages and aspects of kidney diseases, including C3 glomerulopathy (C3G) and other conditions. More results from these studies are expected to be presented at future medical conferences.

The results from the APPLAUSE-IgAN study are promising, marking an important step forward in the management of IgAN, a chronic kidney disease that lacks effective treatment options. If Fabhalta can consistently reduce proteinuria and slow kidney function decline, it may become a cornerstone treatment, reshaping the standard of care in nephrology.

  clinical trials, efficacy, Fabhalta, FDA Accelerated Approval, IgA nephropathy treatment, kidney disease progression, Novartis, Phase III IgAN study, proteinuria reduction, World Congress of Nephrology 2024