Fabhalta Demonstrates Sustained Efficacy in Phase III APPEAR-C3G Study for C3 Glomerulopathy
核心洞察
Novartis' Fabhalta shows clinically meaningful and sustained proteinuria (搜索) reduction in C3 glomerulopathy (搜索) (C3G) patients after 12 months, according to Phase III APPEAR-C3G study data.
The study reveals that Fabhalta treatment leads to early proteinuria (搜索) reduction, observed as early as 14 days, and maintained over the 12-month study period.
An exploratory analysis suggests improvement in estimated glomerular filtration rate (eGFR) slope upon Fabhalta initiation, compared to patients' historical rapid decline.
Novartis presented 12-month data from the Phase III APPEAR-C3G study at American Society of Nephrology Kidney Week 2024, demonstrating that Fabhalta (iptacopan) provides clinically meaningful and sustained benefits for patients with C3 glomerulopathy (搜索) (C3G). The data confirm that treatment with Fabhalta, in addition to supportive care, resulted in a significant reduction in proteinuria (搜索), observed as early as 14 days and sustained over 12 months.
Sustained Proteinuria Reduction and eGFR Improvement
The APPEAR-C3G study included a 6-month randomized, double-blind treatment period comparing Fabhalta to placebo, followed by a 6-month open-label treatment period where all participants received Fabhalta. Results from the open-label period indicated that participants who switched to Fabhalta also experienced proteinuria (搜索) reduction. Furthermore, a pre-specified exploratory analysis suggested an improvement in the estimated glomerular filtration rate (eGFR) slope following Fabhalta initiation, compared to the rapid decline observed historically in these patients.
Favorable Safety Profile
The study also highlighted Fabhalta's favorable safety profile, with no new safety signals identified during the trial. This is particularly important given the chronic nature of C3G and the need for long-term treatment options.
Context of C3 Glomerulopathy
C3G is a rare and severe form of glomerulonephritis characterized by excessive activation of the alternative complement pathway, leading to kidney damage. Approximately 50% of C3G patients progress to kidney failure within 10 years of diagnosis, often requiring lifelong dialysis or kidney transplantation. The current treatment landscape primarily involves supportive care, highlighting the significant unmet need for targeted therapies that can modify the disease course.
Previous Results
Previously presented data at the 2024 European Renal Association Congress showed a statistically significant and clinically meaningful 35.1% proteinuria (搜索) reduction with Fabhalta compared to placebo at 6 months, on top of supportive care.