Novartis has received the US Food and Drug Administration (FDA) accelerated approval for its selective endothelin A receptor antagonist Vanrafia (atrasentan).
Vanrafia is an oral, once-daily, non-steroidal treatment that complements renin-angiotensin system (RAS) inhibitor, with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.
The drug is indicated for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.
The FDA granted accelerated approval based on interim results from the Phase 3 ALIGN study, which evaluated proteinuria reduction at 36 weeks compared to placebo.
However, Vanrafia has not been validated for slowing kidney function decline in IgAN patients, and continued approval is expected based on clinical benefits from the ongoing study.
The eGFR data, which is expected in 2026, will support traditional FDA approval.
Novartis US president Victor Bultó said: “We are proud to expand the treatment landscape in IgA nephropathy with today’s FDA accelerated approval of Vanrafia.
“IgAN is a heterogenous condition that requires differentiated therapies with unique mechanisms of action, and with our multi-asset kidney disease portfolio, we are well positioned to support a broad patient population and advance care for this disease.
“Building on our longstanding legacy in nephrology, we continue to rapidly grow our capabilities in this space. Each launch enables us to more effectively reach patients with the most suitable treatment option and deliver on our promise to transform kidney disease care.”
IgAN is a progressive kidney disease characterised by immune system attacks on the kidneys, leading to glomerular inflammation and proteinuria.
In the ALIGN study, patients receiving Vanrafia with a RAS inhibitor showed a statistically significant proteinuria reduction of 36.1% compared to placebo.
The treatment’s effect appeared as early as week six and sustained through week 36, and was consistent across various subgroups, including age, sex, race, and disease types.
The ALIGN study also showed a similar treatment effect in patients taking both an RAS inhibitor and an SGLT2 inhibitor.
Vanrafia showed a safety profile that aligns with its previous data, with adverse events such as peripheral oedema, anaemia, and liver transaminase elevation.
Vanrafia ALIGN Study investigator and steering committee member Richard Lafayette said: “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.
“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.”
