We are delighted to announce that the National Institute for Health and Care Excellence (NICE) has issued final guidance for use of the HIF-2α inhibitor, belzutifan, for the treatment of people with von Hippel-Lindau (VHL) renal cell carcinoma (RCC) who do not need or want surgery. Belzutifan is approved for use by NHS England under a managed access scheme. Belzutifan meets an unmet treatment need for people with VHL disease.

Kidney cancers frequently have mutations in a gene called the von Hippel-Lindau (VHL) gene, resulting in high levels of a protein called hypoxia-inducible factor, or HIF-2α. This results in a number of changes in the cancer cells and their surrounding environment that favour tumour growth. The novel HIF-2α inhibitor, belzutifan, blocks the action of HIF-2α and blocks cancer cell growth.

Belzutifan has shown to be an effective treatment for Von Hippel-Lindau disease (VHL), a rare condition which affects one in 33,000 people. Von Hippel-Lindau disease is associated with inherited alterations in the VHL gene. It results in benign and malignant tumours in the brain and spinal cord, pancreas or kidney. Up to 70% of patients with VHL develop RCC during their lifetime. Currently, VHL kidney cancer is managed with surgery; tumours less than 3 cm in diameter are removed to limit the risk of spread of the cancer and to avoid a decline in kidney function that results from multiple tumours in the kidney.

The approval was based on the results from a phase 2 study, were 64% of RCC patients responded to treatment with belzutifan.

Belzutifan is also in clinical trials for the treatment of advanced RCC and other types of cancer. There is an ongoing phase 3 clinical trial to compare belzutifan to everolimus in heavily pretreated patients and a combination with cabozantinib as a first-line treatment for advanced clear cell RCC.

Action Kidney Cancer would like to thank those patients and clinicians who contributed to the NICE belzutifan appraisal – without your dedication and perseverance on behalf of the VHL community, the decision might not have been so positive – a BIG thank you!

Read the final guidance on the NICE website here

There is also information for the public, which can be accessed here.