FDA Accelerates Approval for Rare Kidney Disease

The U.S. Food and Drug Administration (FDA) has granted accelerated approval for Novartis’ Vanrafia (atrasentan) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. IgAN is a rare, chronic autoimmune disease that attacks the kidneys. Up to 50 percent of patients with persistent proteinuria, or protein in the urine, progress to kidney failure within 10 to 20 years of diagnosis.

Developed by Novartis, Vanrafia is a selective endothelin A (ETA) receptor antagonist. ETA receptor activation causes proteinuria, which is usually one of the first clinical signs of IgAN. It is a once-daily, non-steroidal, oral treatment that can be added onto supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor. “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,” said Richard Lafayette, MD, professor of medicine, nephrology and director of the Glomerular Disease Center at Stanford University Medical Center, and a Vanrafia ALIGN Study Investigator and Steering Committee Member.

Vanrafia was granted accelerated approval based on a prespecified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared with placebo. In the study, investigators found that patients receiving Vanrafia in combination with a RAS inhibitor achieved a clinically meaningful and statistically significant proteinuria reduction of 36.1 percent. Adverse events reported in greater than or equal to two percent of patients treated with Vanrafia, and more frequently than placebo, include peripheral edema, anemia and liver transaminase elevation. 

The ALIGN study continues to evaluate whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 136. The eGFR data are expected in 2026 and intended to support traditional FDA approval.