Novartis Secures FDA Approval for Vanrafia® to Treat Primary IgA Nephropathy
Thursday, April 03, 2025
Novartis has received accelerated approval from the US Food and Drug Administration (FDA) for Vanrafia® (atrasentan), a selective endothelin A (ETA) receptor antagonist, for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of rapid disease progression.
IgAN is a progressive autoimmune kidney disease that causes inflammation and damage to the glomeruli, leading to proteinuria. It is one of the most common autoimmune kidney diseases, affecting approximately 13 individuals per million annually in the US.
Many patients with persistent proteinuria may develop kidney failure within 10 to 20 years of diagnosis, often requiring dialysis or a kidney transplant. The availability of targeted therapies with different mechanisms of action provides physicians with additional options for managing the condition.
Vanrafia has shown a favourable safety profile, with commonly reported adverse effects including peripheral oedema, anaemia, and elevated liver enzyme levels. Due to the potential risk of liver-related side effects, liver enzyme testing is recommended before and during treatment as needed.
The drug does not require a Risk Evaluation and Mitigation Strategy (REMS) programme but should be avoided in pregnancy due to the risk of birth defects.
The condition is typically identified by a urine protein-to-creatinine ratio (UPCR) of at least 1.5 g/g. Vanrafia is a once-daily, non-steroidal oral treatment that can be added to existing supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.
The approval is based on interim results from the Phase III ALIGN study, which evaluated Vanrafia’s effectiveness in reducing proteinuria at 36 weeks compared to a placebo. While the drug has been shown to lower protein levels in urine, its long-term impact on kidney function decline has not yet been confirmed.
Continued FDA approval will depend on further data from the ongoing ALIGN study, particularly results assessing the decline in estimated glomerular filtration rate (eGFR) over 136 weeks.
These findings are expected in 2026 and will be reviewed to support full FDA approval.
Source: novartis.com
