FDA Approves Belzutifan For Advanced Kidney Cancer

The US Food and Drug Administration (FDA) has recently granted approval for belzutifan, an HIF-2α inhibitor, for the treatment of advanced kidney cancer. This approval specifically applies to patients who have previously undergone immune checkpoint inhibitors or anti-angiogenic therapies. The decision is based on the findings of the phase 3 LITESPARK-005 trial, led by Toni K. Choueiri, MD, and his team at the Dana-Farber Cancer Institute.

The LITESPARK-005 study revealed a substantial reduction in the risk of progression of clear cell renal cell carcinoma, the most commonly diagnosed form of kidney cancer. In comparison with the alternative treatment, everolimus, belzutifan demonstrated a 26% decrease in progression risk and a sixfold increase in treatment response. Initially approved for patients with Von Hippel-Landau disease–associated renal cell carcinoma, belzutifan’s expanded approval now extends its benefits to a broader range of renal cell carcinoma patients beyond the rare subtype.

Dr Choueiri, director of the Lank Center for Genitourinary Cancer at Dana-Farber Cancer Institute, expressed enthusiasm about the approval, stating, “This is exciting news for our patients as it provides a new option for those with refractory kidney cancer.” Describing belzutifan as an oral medication with a unique mechanism of action, Dr Choueiri highlighted its capacity to reduce the risk of disease progression or death while enhancing the quality of life in this patient population when compared to everolimus.

The LITESPARK-005 study enrolled approximately 746 patients who had previously undergone immune checkpoint inhibitor and anti-angiogenic therapy treatments. Through random assignment, patients received either belzutifan or everolimus, allowing researchers to make a comprehensive comparison. The duration of treatment for participants in this study spanned 27.5 months. Belzutifan exhibited a significantly higher response rate than everolimus at 22% vs 3.5%, respectively. Additionally, belzutifan demonstrated strong safety and efficacy levels, reducing the likelihood of treatment discontinuation due to side effects.

This FDA approval represents a promising development for patients battling kidney cancer, particularly those with clear cell renal cell carcinoma. The availability of belzutifan as an alternative option holds particular significance for patients unable to tolerate everolimus, offering expanded treatment possibilities in the treatment of advanced kidney cancer.

Source: 

FDA approves kidney cancer therapy after Dana-Farber-led research shows improved outcomes for patients with advanced disease. Dana-Farber Cancer Institute. December 15, 2023. Accessed February 20, 2024. https://www.dana-farber.org/newsroom/news-releases/2023/fda-approves-kidney-cancer-therapy-after-dana-farber-led-research-shows-improved-outcomes-for-patients-with-advanced-disease