Novartis has announced positive results from the Phase III APPEAR-C3G study, indicating that Fabhalta (iptacopan), when combined with supportive care, delivered sustained benefits for patients with 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.
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The APPEAR-C3G trial is a Phase III, multi-centre, 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.
This treatment for C3G has shown promising results in reducing proteinuria and improving kidney function.
The data was presented at the American Society of Nephrology (ASN) Kidney Week 2024.
During the trial, patients treated with Fabhalta experienced a significant reduction in proteinuria as early as two weeks, with the effect sustained over one year.
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By GlobalDataThis improvement was also observed in patients who transitioned to Fabhalta during the open-label phase of the study.
Moreover, exploratory analysis revealed an improvement in the estimated glomerular filtration rate (eGFR) slope, which is a pivotal measure to demonstrate kidney functioning upon Fabhalta initiation, contrasting with the historical rapid decline in patients.
The safety profile of Fabhalta remained favourable.
Novartis cardiovascular, renal and metabolism development unit global head David Soergel said: “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.
“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.”
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).
