Novartis has announced sustained, clinically meaningful results of oral Fabhalta (iptacopan) from the Phase 3 APPEAR-C3G study of patients with C3 glomerulopathy (C3G).
The APPEAR-C3G study assessed the efficacy and safety of twice-daily oral Fabhalta in adults with C3G. It included a six-month, randomised, double-blind phase comparing Fabhalta to placebo, followed by a six-month open-label phase, where all participants received Fabhalta.
The data revealed that Fabhalta, when combined with supportive care, delivered clinically meaningful and sustained results for patients with C3G over 12 months.
Treatment with iptacopan led to a clinically meaningful reduction in proteinuria, noticeable as early as 14 days and sustained at 12 months.
In the open-label phase, participants switching to Fabhalta also showed a reduction in proteinuria. The study showed a statistically significant and clinically meaningful 35.1% reduction in proteinuria compared to placebo at six months.
Additionally, an improvement in the estimated glomerular filtration rate (eGFR) slope was observed after starting Fabhalta, compared to a historic rapid decline.
In addition, iptacopan demonstrated a favourable safety profile, with no new safety signals reported.
Novartis renal and metabolism development unit cardiovascular 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 European Union (EU), China, and Japan, with an expectation for submission in the US by year-end.
Fabhalta received US Food and Drug Administration (FDA) approval in December 2023 for treating adults with paroxysmal nocturnal haemoglobinuria (PNH).
It also secured accelerated approval in August 2024 for reducing proteinuria in specific adults with primary IgA nephropathy (IgAN).
Iptacopan is also being investigated for various rare kidney diseases, including C3G, atypical haemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN).
Additionally, the Swiss drugmaker is advancing two other IgAN therapies, atrasentan and zigakibart.
