FDA Approves Fabhalta for Treatment of Proteinuria in IgAN

The US Food and Drug Administration (FDA) has granted accelerated approval of Novartis’ drug Fabhalta (iptacopan) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.

IgAN occurs when clumps of antibodies build up in the kidneys, resulting in inflammation that damages the glomeruli, making it more difficult for the kidneys to filter out waste. Fabhalta is an inhibitor of the alternative complement pathway, the activation of which is thought to contribute to the pathogenesis of IgAN.

The accelerated approval was based on prespecified interim analysis of the phase 3 APPLAUSE-IgAN trial, which found that Fabhalta reduced proteinuria by 43.8% compared with a placebo. Continued approval may be contingent on verification and description of clinical benefit from the ongoing study, evaluating whether Fabhalta slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline over 24 months. The eGFR data should be available upon completion of the study next year.

The approval allows Fabhalta to compete with drug maker Calliditas’ Tarpeyo and Travere Therapeutics’ Filspari. Meanwhile, Novartis is developing two other drugs, atrasentan and zigakibart, to treat IgAN. Other drug makers, including Otsuka and Vera Therapeutics, are also working on IgAN treatments.