FDA Grants Accelerated Approval to Vanrafia for IgAN, a Rare Kidney Disease

The 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.

Primary immunoglobulin A nephropathy (IgAN) is a rare, chronic autoimmune disease that attacks the kidneys. Up to 50% of patients with persistent proteinuria, or protein in the urine, progress to kidney failure within 10 to 20 years of diagnosis.

Vanrafia, developed by Novartis, 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.

Richard Lafayette, M.D.

“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,” Richard Lafayette, M.D., professor of medicine, nephrology, and director of the Glomerular Disease Center at Stanford University Medical Center, said in a news release. Lafayette is a Vanrafia ALIGN Study Investigator and Steering Committee Member.

Vanrafia has a wholesale acquisition cost of $162,500 annually. Eligible privately insured patients may receive Vanrafia for free for up to 12 months while coverage is being pursued and may also pay as little as $0 for Vanrafia through Novartis’ $0 Co-Pay Plus offer. A spokesperson said the Novartis Patient Support provides personalized support to assist patients in navigating their insurance coverage and identifying financial assistance options.

Although healthcare providers can start to prescribe Vanrafia right away, Novartis anticipates the product will be available for specialty pharmacies to dispense within the next few weeks, the Novartis spokesperson said.

Vanrafia was granted accelerated approval based on a prespecified interim analysis of the phase 3 ALIGN study measuring the reduction of proteinuria at 36 weeks compared with placebo.

Investigators found that patients receiving Vanrafia in combination with a RAS inhibitor achieved a clinically meaningful and statistically significant proteinuria reduction of 36.1%. Adverse events reported in ≥2% of patients treated with Vanrafia, and more frequently than placebo, include peripheral edema, anemia, and liver transaminase elevation. Vanrafia may cause serious birth defects. Vanrafia does not require a REMS program.

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.

Related: FDA Approves Novartis’ Oral Fabhalta as First Treatment for C3G

Novartis has been building a portfolio of therapies for kidney disease. Fabhalta was granted FDA approval in C3 glomerulopathy (C3G) in March 2025 and accelerated approval in IgAN in August 2024. Fabhalta is also being studied in other kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN).

Additionally, an investigational subcutaneous monoclonal antibody, zigakibart, is currently in phase 3 development in IgAN, with results expected in 2026.