Novartis has announced positive results from the Phase III APPEAR-C3G study, revealing that Fabhalta (iptacopan), when used with supportive care, provided sustained benefits for patients suffering from C3 glomerulopathy (C3G) over a 12-month period. C3G is a severe kidney disease that often leads to kidney failure within ten years of diagnosis, highlighting the urgent need for effective treatments.
The APPEAR-C3G trial is a Phase III, multi-center, parallel-group, randomised, double-blind, placebo-controlled study designed to assess the safety and efficacy of Fabhalta, the only oral Factor B inhibitor targeting the alternative complement pathway.
Significant Reduction in Proteinuria
Data presented at the American Society of Nephrology (ASN) Kidney Week 2024 showed that patients treated with Fabhalta experienced a significant reduction in proteinuria as early as two weeks, with the effect sustained over one year. This improvement was also observed in patients who transitioned to Fabhalta during the open-label phase of the study.
Improvement in Kidney Function
Exploratory analysis revealed an improvement in the estimated glomerular filtration rate (eGFR) slope, a key measure of kidney function, upon Fabhalta initiation, contrasting with the historical rapid decline in patients with C3G. The safety profile of Fabhalta remained favorable throughout the study.
Regulatory Submissions and Future Plans
Regulatory submissions for Fabhalta in C3G have been completed in the EU, China, and Japan, with a submission expected in the US by the end of the year. Fabhalta has already been approved by the FDA in December 2023 for treating adults with paroxysmal nocturnal haemoglobinuria and received accelerated approval in August 2024 to reduce proteinuria in adults with primary IgA nephropathy (IgAN).
David Soergel, Global Head of the Cardiovascular, Renal and Metabolism Development Unit at Novartis, stated, “We are thrilled to share these data, which demonstrate the potential of Fabhalta in C3G, and look forward to working with regulatory authorities with the goal of bringing this innovative medicine to this patient community.” He added, “Building on the longstanding experience of Novartis in nephrology and our first rare kidney disease approval in IgA nephropathy earlier this year, these results in C3G show continued advancement of our broad, industry-leading portfolio, which aims to transform care for these patients.”
