Novartis presented 12-month data from the Phase III APPEAR-C3G study at the American Society of Nephrology Kidney Week 2024, demonstrating that Fabhalta (iptacopan) provides clinically meaningful and sustained benefits for patients with C3 glomerulopathy (C3G). The study's findings highlight the potential of Fabhalta, when added to supportive care, to significantly improve outcomes for individuals affected by this rare and severe kidney disease. Approximately 50% of C3G patients progress to kidney failure within 10 years of diagnosis, at which point they require lifelong dialysis and/or kidney transplantation.
Sustained Proteinuria Reduction
The APPEAR-C3G study's 12-month data confirm that treatment with Fabhalta resulted in clinically meaningful proteinuria reduction, a key indicator of kidney damage. This reduction was observed as early as 14 days after treatment initiation and was sustained throughout the 12-month study period. Furthermore, in an open-label extension of the study, patients who were switched to Fabhalta also experienced a reduction in proteinuria, reinforcing the drug's efficacy.
Improvement in eGFR Slope
An exploratory analysis of the data revealed a potential improvement in the estimated glomerular filtration rate (eGFR) slope following the start of Fabhalta treatment. This suggests that Fabhalta may help to slow the progression of kidney damage in C3G patients, potentially delaying or preventing the need for dialysis or kidney transplantation. The eGFR slope improvement was compared to patients’ historic rapid decline.
Safety Profile
Fabhalta demonstrated a favorable safety profile throughout the APPEAR-C3G study, with no new safety signals identified during the 12-month treatment period. This is an important consideration for patients with C3G, who often require long-term treatment to manage their condition.
Study Design
The APPEAR-C3G study was a Phase III trial designed to evaluate the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G. The study included a 6-month randomized, double-blind treatment period, during which patients received either Fabhalta or a placebo in addition to supportive care. This was followed by a 6-month open-label treatment period, where all participants received Fabhalta. Results previously presented at the 2024 European Renal Association Congress demonstrated a statistically significant and clinically meaningful 35.1% proteinuria reduction vs. placebo on top of supportive care at 6 months.
