The U.S. FDA has granted accelerated approval to Novartis' complement inhibitor, Fabhalta (iptacopan), for the treatment of primary immunoglobulin A nephropathy (IgAN).
Fabhalta is now the only complement inhibitor approved for reducing proteinuria in adults at risk of rapid disease progression, defined by a urine protein-to-creatinine ratio of ≥1.5 g/g. The drug targets the alternative complement pathway, which is believed to contribute to the pathogenesis of IgAN by being overly activated in the kidneys.
The approval is based on interim results from the phase 3 APPLAUSE-IgAN study, which demonstrated that Fabhalta achieved a 44% reduction in proteinuria from baseline at nine months, compared to a 9% reduction in the placebo group. The approval is contingent on confirmatory trials currently being conducted in the ongoing phase 3 APPLAUSE-IgAN study.
Despite current treatments, up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis. Novartis is also advancing other therapies for IgAN, including atrasentan and zigakibart, which are in late-stage development.
Fabhalta snagged its first FDA approval last December, becoming the first oral monotherapy for the treatment of adults with the rare, chronic blood disorder, paroxysmal nocturnal hemoglobinuria (PNH).
