The Food and Drug Administration (FDA) in the United States has approved a new treatment called belzutifan for people with advanced kidney cancer who have failed treatment with immunotherapy and/or tyrosine kinase inhibitors and their cancer is getting worse.

Studies looking at the genes of people with kidney cancer have shown changes (mutations) in a gene called the von Hippel-Lindau gene (VHL). This results in high levels of a protein called hypoxia-inducible factor, or HIF-2α in the blood of these patients. This causes changes in the cancer cells that make the tumour grow. A new medicine called belzutifan is a tablet that blocks the action of HIF-2α and, therefore, blocks cancer cell growth.

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 tyrosine kinase inhibitor therapy. After 1 and a half years of follow-up, nearly a quarter of the patients who were taking belzutifan did not have growth of their cancer compared to 1 in 10 of the patients taking everolimus.

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. In nearly a quarter of patients on belzutifan their cancer shrank or remained stable, compared to nearly 4 in 100 patients on everolimus. nearly 4 in 100 patients had a complete response to treatment and their cancer disappeared on belzutifan treatment. The average length of response was longer with belzutifan at just over a year and a half compared to just over a year with everolimus.

Currently, belzutifan is approved in the United States and Scotland 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 will be appraised next year by NICE and the SMC for use on the NHS for advanced kidney cancer that has already been treated with an anti-cancer medication.

Read more in Targeted Oncology here