Novartis said that Fabhalta (iptacopan) has shown a significant reduction in proteinuria in the Phase 3 APPEAR-C3G study in adult patients with C3 glomerulopathy (C3G).
Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway.
It was approved by the US Food and Drug Administration (FDA) in late 2023 and the European Medicines Agency (EMA) earlier this month. Both approvals are for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
The multicentre, randomised APPEAR-C3G study evaluated twice-daily oral Fabhalta (200mg) in C3G patients against a placebo on top of supportive care.
As per the six-month, double-blind period results, patients treated with Fabhalta along with supportive care achieved a 35.1% reduction in proteinuria, as measured by 24-hour urine protein to creatinine ratio (UPCR).
Further information regarding the secondary endpoint of the estimated glomerular filtration rate (eGFR) revealed a slight enhancement of +2.2 mL/min/1.73 m2 over six months with Fabhalta compared to a placebo.
Additionally, the trial demonstrated that Fabhalta exhibited a favourable safety profile with no new safety concerns.
Novartis cardiovascular, renal and metabolism development unit global head David Soergel said: “Our ambition is to transform the care of patients living with rare kidney diseases by discovering, developing and delivering innovative treatment options.
“The APPEAR-C3G results add to the growing body of evidence demonstrating Fabhalta’s potential to target the underlying pathophysiological drivers and to provide clinically meaningful outcomes in a range of rare conditions.”
The APPEAR-C3G study is ongoing, with an additional six-month phase where all participants, including those previously on placebo, are administered Fabhalta in an open-label period.
Novartis is planning to submit regulatory applications, including to the FDA and EMA, for the adult C3G indication during the latter half of 2024.
In a separate development, the Swiss drugmaker announced pre-specified interim analysis of atrasentan, an investigational, potent, and selective oral ETA receptor antagonist, in the Phase 3 ALIGN study of patients with IgA nephropathy (IgAN).
Patients receiving atrasentan, alongside supportive care experienced clinically meaningful reduction of 36.1% in proteinuria at 36 weeks, against those on placebo along with supportive care.
