A new treatment has been approved by the FDA for adults suffering from a serious kidney disorder known as primary immunoglobulin A nephropathy (IgAN), also called Berger’s disease. This is an autoimmune condition where the immune system mistakenly attacks the kidneys, causing inflammation and potential long-term damage. IgAN occurs when immunoglobulin A (IgA) antibodies accumulate in the kidneys, leading to the breakdown of kidney tissues. The disorder is known to cause symptoms such as blood or protein in the urine, swelling in the hands and feet, and high blood pressure. Over time, if untreated, it can progress to kidney failure, necessitating dialysis or a kidney transplant.
The newly approved drug, Vanrafia (atrasentan), is a once-daily pill that works by targeting and blocking a specific receptor in the kidneys known as the endothelin A (ETA) receptor. This receptor plays a key role in causing inflammation and damage to kidney cells, which leads to proteinuria, or excess protein in the urine. Proteinuria is a significant marker of kidney damage and a key risk factor for kidney failure. Vanrafia is the first drug specifically designed to reduce proteinuria in patients with IgAN, offering a new avenue for treatment in a condition that has previously been managed with supportive care.
The approval of Vanrafia by the FDA was based on the results of a 36-week clinical trial involving 340 patients with IgAN and proteinuria who were at high risk for kidney damage. In the trial, patients who took Vanrafia in combination with one standard medication showed a 36.1% reduction in proteinuria, while those on Vanrafia combined with two standard medications experienced a slightly greater reduction of 37.4%. These improvements in proteinuria were observed as early as six weeks into the treatment and continued through the 36-week mark, demonstrating the drug’s effectiveness over time.
This approval marks a significant milestone for those living with IgAN, as it provides an option to address a key aspect of the disease—proteinuria. According to experts, early intervention to reduce proteinuria is crucial for improving long-term outcomes for IgAN patients, many of whom may otherwise progress to kidney failure. The treatment offers hope to individuals who have had limited options in the past.
Vanrafia was found to be generally well tolerated in the clinical trial, with no new safety concerns raised. However, there are some potential side effects associated with the drug. Common side effects include swelling in the hands, legs, ankles, and feet, as well as anemia and increased liver enzyme levels. Because Vanrafia can impact liver function, doctors will monitor liver health before and during treatment. Additionally, patients taking Vanrafia should inform their healthcare provider about their medical history and any other medications, including over-the-counter drugs, vitamins, and herbal supplements, as these can interfere with the drug’s effectiveness or exacerbate side effects.
The drug also has significant precautions for women of childbearing age, as it can cause serious birth defects. Women must have a negative pregnancy test before starting Vanrafia and use effective birth control during treatment and for two weeks after stopping the medication. It is also advised that patients do not breastfeed while taking Vanrafia. In men, Vanrafia may affect sperm count and fertility, so men considering treatment should discuss potential fertility concerns with their doctor if having children is a priority.
The introduction of Vanrafia offers a promising new treatment option for those suffering from IgAN, addressing an unmet need in the management of the disease. With ongoing monitoring and careful management of side effects, the drug could significantly improve the quality of life for those living with this debilitating kidney disorder.
