Novartis has announced that the US Food and Drug Administration (FDA) has granted traditional approval to Fabhalta® (iptacopan) for slowing kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. The decision follows the drug's accelerated approval in August 2024, which was based on its ability to reduce proteinuria.
Fabhalta is the first and only complement inhibitor approved to significantly slow kidney function decline in patients with primary IgAN. The approval was granted under the FDA's priority review process and is supported by results from the Phase III APPLAUSE-IgAN clinical trial.
The study demonstrated that Fabhalta slowed the decline in kidney function by 48% compared with placebo over a two-year period. Patients receiving the treatment showed an annualized estimated glomerular filtration rate (eGFR) decline of -3.0 mL/min/1.73 m²/year, compared with -5.7 mL/min/1.73 m²/year in the placebo group. The medicine also produced clinically meaningful reductions in proteinuria as early as two weeks after treatment initiation, with benefits sustained throughout the study.
IgA nephropathy is one of the most common autoimmune kidney diseases worldwide. Without effective treatment, many patients with persistent proteinuria progress to kidney failure within 10 to 20 years, often requiring dialysis or kidney transplantation.
Fabhalta is an oral Factor B inhibitor that selectively blocks the alternative complement pathway, a major driver of inflammation and kidney damage in IgAN. By targeting this underlying disease mechanism, the therapy aims to preserve kidney function and delay disease progression.
The safety profile of Fabhalta was consistent with previous clinical studies. The most commonly reported adverse events included abdominal pain, dizziness, and nausea. Due to the potential risk of serious infections caused by encapsulated bacteria, the medicine is available only through a Risk Evaluation and Mitigation Strategy (REMS) program, which requires appropriate vaccinations before treatment.
Novartis stated that the approval marks an important milestone in expanding treatment options for patients with IgAN. The company is also advancing its broader kidney disease portfolio, which includes Vanrafia® (atrasentan) and the investigational therapy zigakibart, as part of its commitment to improving long-term outcomes for patients with chronic kidney diseases.
