Novartis oral Fabhalta win in Phase III C3 glomerulopathy trial

Novartis (NOVN: VX) yesterday presented 12-month data from the Phase III APPEAR-C3G study at American Society of Nephrology (ASN) Kidney Week 2024 showing that patients with C3 glomerulopathy (C3G) treated with oral Fabhalta (iptacopan) in addition to supportive care experienced clinically meaningful, sustained results at one year.

These data confirm treatment with Fabhalta resulted in clinically meaningful proteinuria reduction, which was seen as early as 14 days, and sustained at 12 months, the Swiss pharma giant said. Similarly, in an open-label period of the study, proteinuria reduction was seen in participants who were switched to Fabhalta. In addition, improvement in estimated glomerular filtration rate (eGFR) slope was observed upon Fabhalta initiation compared to patients’ historic rapid decline based on results from a prespecified exploratory analysis. Fabhalta showed a favorable safety profile, with no new safety signals.1

The APPEAR-C3G study evaluated the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G. The study was comprised of a six-month randomized, double-blind treatment period with Fabhalta compared to placebo, followed by an additional six-month open-label treatment period where all participants received Fabhalta. Results previously presented at the 2024 European Renal Association (ERA) Congress demonstrated a statistically-significant and clinically-meaningful 35.1% proteinuria reduction versus placebo on top of supportive care at six months.