Novartis Receives USFDA Approval for Vanrafia to Treat Berger’s Disease

Published: 07 Apr 2025

Author: Precedence Research

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Vanrafia, a Novartis drug, has been approved by the FDA to treat primary immunoglobulin A nephropathy (IgAN), or Berger's disease. IgAN is a rare and progressive kidney disease that affects thousands in the US. The disease is autoimmune, usually involving an immune attack on the kidney with resulting inflammation and impregnation. The occurrence of IgAN in the US is approximately 13 per million people annually and varies from individual to individual, with some experiencing mild symptoms and others progressing within short periods to kidney failure.

The Approval of Vanrafia: A Promising Advancement

Vanrafia is a new drug that can be given orally as a daily dose. It acts as a non-steroidal medicine and works on the patients in combination with other treatments for their illnesses. It is also used in conjunction with an RAS inhibitor, with or without an SGLT2 inhibitor. The choice of Novartis for accelerating the way toward quick approval hung on interim results from a Phase III ALIGN study sounding the endpoint of proteinuria reduction over 36 weeks compared to placebo. However, to establish efficiency in reducing kidney function decline, Vanrafia has to undergo more tests. The study is currently ongoing in Phase III ALIGN which will determine whether or not Vanrafia actually slows progression through the measurement of eGFR decline at week 136. Results from this important trial are due in 2026, to support conventional FDA approval.

Expert Insights and Patient Impact

The approval of Vanrafia, a selective ETA receptor antagonist, has been congratulated by Dr. Richard Lafayette, Professor of Medicine, Nephrology, and Director of the Glomerular Disease Center at Stanford University Medical Center. This URTI reduces proteinuria, one of the main risk factors in IgA nephropathy. It should, in due course, fit in with existing treatment plans for patients without any need for REMS. Early treatment intervention is the key to improving patient outcomes. Bonnie Schneider, Director and Co-Founder of the IgA Nephropathy Foundation had an optimistic take on the approval and the fact that the more treatment options available for the community, the more tailored care available to patients as per the varying impact of the disease.

Novartis’ Broader Kidney Disease Portfolio

Fabhalta which is a new kidney disease-based drug has been approved by FDA. In March 2025, it will be used as an indication for IgAN treatment in August 2024 and for C3 Glomerulopathy. Besides, it will also be researched for rare kidney diseases such as atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis, and lupus nephritis. Novartis is also advancing towards the development of an investigational anti-APRIL monoclonal antibody called zigakibart, which is currently under Phase III investigational trial for patients with IgAN. Results are expected during 2026.

Future Outlook and Anticipated Impact

Deepening its commitment to the kidney disease area, Novartis is set on delivering pioneering therapies for patients suffering rare and often ignored diseases. The approval of Vanrafia has made a considerable advance for IgAN patients and offers hope for a more effective treatment regimen to improve patient outcomes and quality of life. With possible eGFR data slated for 2026, all indications are that Vanrafia's traditional FDA approval is in favorable view. For many patients and their families, this has raised hope once again for possibly better management of a complex life-changing disease.