FDA Approves New Treatment for Serious Kidney Disorder

A new drug has received a quick federal nod for adults with a serious kidney disorder known as primary immunoglobulin A nephropathy (IgAN). The drug is a once-daily nonsteroidal pill that can be taken along with standard supportive care to reduce protein in the urine, known as proteinuria.

IgAN, which is also called Berger disease, is an autoimmune kidney condition where the immune system mistakenly attacks the kidneys. This happens when IgA antibodies build up, causing inflammation and damage over time. Symptoms include blood or protein in the urine, swelling in the hands and feet, and high blood pressure. In severe cases, it can lead to kidney failure, requiring dialysis or a transplant. Treatment involves supportive care, highlighting the need for targeted therapies that can specifically reduce proteinuria and improve outcomes.

The drug, known as atrasentan and sold as Vanrafia, blocks a specific protein in the kidneys known as endothelin A (ETA) receptor, which causes inflammation and cellular damage leading to proteinuria. Vanrafia is the first drug of its kind to reduce proteinuria in patients with IgAN, according to a news release by Novartis, the drug’s maker. 

The FDA’s decision is based on 36-week results from an ongoing clinical trial involving 34src patients with IgAN nephropathy and proteinuria who are at high risk for kidney damage. Patients taking once-daily oral Vanrafia along with one standard medication saw a 36.1% drop in proteinuria compared with those taking placebo, with improvements starting at week 6 and continuing through week 36. A second group using Vanrafia with two standard medications had a similar 37.4% reduction.

The approval marks “an important milestone for people living with IgA nephropathy,” Richard Lafayette, MD, director of the Glomerular Disease Center at Stanford University Medical Center and one of the trial’s lead investigators, said in a statement. “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.”

Vanrafia was generally well tolerated, with no new safety concerns reported. Common side effects include swelling in the hands, legs, ankles, and feet, as well as anemia and increased liver enzyme levels. Since the drug can cause liver issues, doctors will monitor liver function before and during treatment. Patients should also tell their doctor about their medical history and about all the prescription and over-the-counter medications, vitamins, and herbal supplements they take, as these may interfere with the drug’s action or worsen side effects. As Vanrafia can cause serious birth defects, women need to have a negative pregnancy test before starting the medication and use reliable birth control during treatment and for two weeks after stopping it. Patients are advised not to breastfeed when taking Vanrafia. In men, Vanrafia may lower sperm counts and affect their fertility. Patients should inform their doctor if being able to have children is important to them.

SOURCE:

Novartis: “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).”