Novartis receives FDA accelerated approval for Vanrafia (atrasentan), the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary IgA nephropathy (IgAN)

 Ad hoc announcement pursuant to Art. 53 LR

• Vanrafia can be seamlessly added to supportive care in IgAN and used as a foundational therapy with no requirement for a REMS (Risk Evaluation Mitigation Strategy) program¹
  
• Phase III data showed Vanrafia achieved proteinuria reduction of 36.1% (P<0.0001) vs. placebo with improvements seen at Week 6 and sustained through Week 36 and favorable safety^1,2
  
  
• IgAN is a progressive, rare kidney disease; up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis^3-9
  
  
• With third FDA approval in under 1 year across its renal portfolio, Novartis is uniquely positioned to lead a transformation in kidney care

Basel, April 3, 2025 – Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Vanrafia (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g¹. Vanrafia 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^1,2.

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 to placebo¹. It has not been established whether Vanrafia slows kidney function decline in patients with IgAN. The continued approval of Vanrafia may be contingent upon the verification of clinical benefit from the ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 136¹. The eGFR data are expected in 2026 and intended to support traditional FDA approval.

“Today’s approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,” said Richard Lafayette, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member. “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.”