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American Society of Nephrology's Kidney Week 2024 | News | Updates | iPharmaCenter

Novartis presented new data on Fabhalta, showing a significant reduction in proteinuria that was sustained for up to one year


Novartis has shared one-year findings from the Phase III APPEAR-C3G study, presented at the American Society of Nephrology's Kidney Week 2024, highlighting the benefits of Fabhalta (iptacopan) for patients with C3 glomerulopathy (C3G).

Patients taking the oral treatment Fabhalta alongside supportive care demonstrated sustained improvements, showing significant reductions in proteinuria that were first observed at 14 days and maintained over 12 months.

Additionally, the treatment led to a positive shift in the slope of estimated glomerular filtration rate (eGFR), an essential indicator of kidney health, compared to historical declines in these patients. Fabhalta also maintained a favorable safety profile, with no new safety concerns identified.


The APPEAR-C3G trial assessed the effectiveness and safety of twice-daily oral Fabhalta in adults with C3G, beginning with a six-month randomized, double-blind phase comparing Fabhalta to a placebo, followed by a six-month open-label period where all participants received Fabhalta. Earlier findings shared at the 2024 European Renal Association Congress revealed a significant 35.1% reduction in proteinuria for Fabhalta-treated patients over six months compared to placebo.


C3G is a rare kidney disease with limited treatment options, often leading to end-stage kidney failure within a decade, requiring either dialysis or a kidney transplant. Fabhalta is the first oral Factor B inhibitor targeting the alternative complement pathway, offering potential as a novel treatment for C3G in the U.S. Following its December 2023 approval for paroxysmal nocturnal hemoglobinuria (PNH) and conditional approval in August 2024 for primary IgA nephropathy (IgAN), regulatory filings for Fabhalta in C3G are underway in the EU, China, Japan, and anticipated in the U.S. by the end of the year.


Fabhalta, developed by Novartis, is currently being tested across multiple rare kidney diseases, such as atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN), to gather further safety and efficacy data to support potential approvals in these conditions.


About APPEAR-C3G

The APPEAR-C3G study (NCT04817618) is a Phase III, multicenter, randomized, double-blind, placebo-controlled trial focused on evaluating the safety and effectiveness of Fabhalta in patients with C3G. Participants were randomized to receive either Fabhalta or a placebo along with supportive care during the initial six months, followed by a six-month open-label phase where all received Fabhalta. The primary aim was to measure changes in proteinuria from baseline at six months, assessed by the 24-hour urine protein to creatinine ratio (UPCR). A separate cohort is ongoing to assess Fabhalta in adolescent C3G patients.

Throughout the study, most side effects were mild to moderate, with no severe outcomes like meningitis, meningococcal sepsis, or treatment discontinuations.


Understanding C3 Glomerulopathy (C3G)

C3G is an exceptionally rare and progressive kidney disease, primarily affecting children and young adults, with only 1-2 cases diagnosed per million people each year. In C3G, the alternative complement pathway in the immune system becomes overly active, leading to C3 protein deposits within kidney glomeruli—vital structures that help filter blood. This results in chronic kidney damage, proteinuria, blood in the urine, and reduced kidney function over time.

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