Novartis' oral Factor B inhibitor of the alternative complement pathway, Fabhalta, has met its phase 3 goals for treating IgA nephropathy (IgAN).
In the APPLAUSE-IgAN trial, Fabhalta demonstrated statistically significant, clinically meaningful superiority compared to placebo in slowing IgAN progression measured by annualized total slope of estimated glomerular filtration rate (eGFR) decline over two years.
Fabhalta received accelerated approval last year to treat adults with IgAN by reducing proteinuria. Novartis is now seeking to secure traditional FDA approval for IgAN, using the phase 3 data to support its 2026 submission.
A progressive autoimmune kidney disease, IgAN often leads to kidney failure. Compared to a placebo, Fabhalta showed a significant, clinically-meaningful improvement in estimated glomerular filtration rate (eGFR) slope, which is a key marker of kidney function. It is the first complement inhibitor for adults with IgAN to receive approval.
Fabhalta was originally approved in 2023 as a treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria. It was approved in March 2025 as a treatment for complement 3 glomerulopathy.