Novartis has announced positive 12-month results from the Phase III APPEAR-C3G study, demonstrating the sustained efficacy and safety of Fabhalta (iptacopan) in patients with C3 glomerulopathy (C3G). The data, presented at the American Society of Nephrology (ASN) Kidney Week 2024, indicate that Fabhalta, when used with supportive care, provides clinically meaningful benefits to C3G patients over a one-year period.
The study confirmed that Fabhalta treatment led to a significant reduction in proteinuria, a key indicator of kidney damage. This reduction was observed as early as 14 days after treatment initiation and was maintained throughout the 12-month study duration. Furthermore, an exploratory analysis revealed an improvement in the estimated glomerular filtration rate (eGFR) slope upon starting Fabhalta, contrasting with the rapid historical decline typically seen in these patients.
Proteinuria Reduction
The primary endpoint of the APPEAR-C3G study focused on the change in proteinuria levels. The results showed a statistically significant and clinically relevant reduction in proteinuria in patients treated with Fabhalta compared to baseline. The open-label extension of the study further supported these findings, with patients who switched to Fabhalta also experiencing a notable decrease in proteinuria.
eGFR Improvement
Estimated glomerular filtration rate (eGFR) is a critical measure of kidney function. The study's exploratory analysis indicated that Fabhalta treatment was associated with an improved eGFR slope, suggesting a potential slowing of disease progression. This is particularly significant given the typically rapid decline in kidney function observed in C3G patients.
Safety Profile
Fabhalta exhibited a favorable safety profile throughout the study. No new safety signals were identified, reinforcing the drug's tolerability in this patient population. This is an important consideration for long-term treatment of a chronic condition like C3G.
These findings suggest that Fabhalta could represent a significant advancement in the treatment of C3G, addressing a critical unmet need for patients with this rare and progressive kidney disease.
