Novartis presents latest Phase III data on Fabhalta

Swiss pharma giant Novartis (NOVN: VX) advanced its rare kidney disease portfolio with results from the six-month, double-blind period of the Phase III APPEAR-C3G study of Fabhalta (iptacopan) at the European Renal Association (ERA) Congress.

Patients treated with Fabhalta in addition to supportive care achieved a 35.1% (p=0.0014) reduction in proteinuria (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 6 months when compared to placebo on top of supportive care. In many kidney diseases, proteinuria reduction is an increasingly recognized surrogate marker correlating with delaying progression to kidney failure.

Fabhalta, which was approved by the US Food and Drug Administration (FDA) in December 2023 and the European Medicines Agency (EMA) in May 2024 for the treatment of adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), is an oral Factor B inhibitor of the alternative complement pathway being investigated in adult patients with C3 glomerulopathy (C3G).