Novartis’ Vanrafia (atrasentan) has been granted accelerated approval by the US Food and Drug Administration (FDA) to treat patients with the rare kidney disease immunoglobulin A nephropathy nephropathy (IgAN).
The non-steroidal treatment has been specifically authorised to reduce excess protein in the urine (proteinuria) of adults with primary IgAN who are at risk of rapid disease progression.
Almost 13 out of every million people in the US are diagnosed each year with IgAN, which occurs when IgA accumulates in the kidneys and damages them. This impairs their filtering function and, as a result, the kidneys start to let substances such as blood and protein leak into the urine.
Despite current treatments, up to half of IgAN patients with persistent proteinuria progress to kidney failure within ten to 20 years of diagnosis.
Taken orally once daily, Vanrafia is an endothelin A (ETA) receptor antagonist that can be added to supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.
The FDA’s decision on the drug was based on positive results from the late-stage ALIGN study, in which patients receiving Vanrafia in combination with a RAS inhibitor achieved clinically meaningful and statistically significant proteinuria reduction of 36.1% compared to placebo, with results observed as early as week six and sustained to week 36.
Continued approval of Vanrafia may be contingent on the verification of clinical benefit in ALIGN, which is evaluating whether the drug slows disease progression as measured by estimated glomerular filtration rate decline at week 136.
ALIGN study investigator and steering committee member, Richard Lafayette, Stanford University Medical Center, said: “[This] approval marks an important milestone for people living with IgAN, offering a new option that can be seamlessly integrated into their existing treatment plan… 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.”
The approval comes just two weeks after Novartis’ oral Fabhalta (iptacopan) was approved by the FDA as the first treatment for the ultra-rare kidney disease complement 3 glomerulopathy. Fabhalta was also approved by the US regulator for IgAN in August.
