Kidney cancers often have mutations in a gene called the von Hippel-Lindau (VHL) gene. This results in high levels of a protein called hypoxia-inducible factor, or HIF-2α. HIF-2α causes several changes in the cancer cells that cause the tumour to grow. Belzutifan is a HIF-2α inhibitor, which blocks the action of HIF-2α.

In a recent phase 3 clinical trial, called the LITESPARK-005 trial, belzutifan treatment significantly reduced the risk of the cancer getting worse (progressing) compared to everolimus in patients with advanced kidney cancer who had failed up to 3 previous treatments with immunotherapy and/or targeted therapy.

Overall survival time was also improved in the patients taking belzutifan, although the difference between belzutifan and everolimus was not clinically significant.

Belzutifan also improved response to treatment and was relatively well tolerated.

Currently, belzutifan is approved in the United States for patients with von Hippel-Lindau disease and kidney tumours, brain or spinal cord tumours, or pancreatic tumours that do not require immediate surgery. Belzutifan is also currently being appraised by NICE and the SMC for use on the NHS for VHL-associated kidney cancer.

Read more in Urology Times here