The US Food and Drug Administration (FDA) has granted accelerated approval to Novartis’ Vanrafia, a treatment aimed at decreasing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.
The non-steroidal selective endothelin A (ETA) receptor antagonist is tailored for one-time-a-day oral administration and can be used alongside other supportive care options such as renin-angiotensin system (RAS) inhibitor, with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.
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FDA’s accelerated approval is supported by a prespecified interim analysis from the ongoing randomised Phase III ALIGN trial, which showed proteinuria minimisation at 36 weeks against a placebo.
The trial assessed the safety and efficacy of the therapy against placebo in patients with IgAN.
Subjects treated with the therapy in conjunction with a RAS inhibitor experienced a reduction of 36.1% in proteinuria, with improvements observed as early as week six and maintained through to week 36.
Continued approval of the therapy is dependent on clinical benefit verification from the trial, assessing if the therapy slows the progression of disease according to a measure of estimated glomerular filtration rate (eGFR) decline data at week 136. This data is anticipated in 2026.
Vanrafia ALIGN study investigator and Stanford University Medical Center Glomerular Disease Center director Professor Richard Lafayette stated: “Vanrafia is a selective ETA receptor antagonist that effectively reduces proteinuria, a major risk factor in IgAN. Taking early, decisive action is critical to help improve outcomes for these patients who too often progress toward kidney failure.”
A rare, progressive kidney disease, IgAN leads to glomerular inflammation and proteinuria, resulting from the immune system’s attack on the kidneys.
Vanrafia’s accelerated approval is the third in the US for the company’s kidney disease portfolio, following the agency’s approvals for Fabhalta in C3 glomerulopathy in March 2025 and another accelerated approval for IgAN in August 2024.
