We are delighted to announce that the Scottish Medicines Consortium (SMC) has approved the use of the hypoxia-inducible factor-2 alpha (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 Scotland under a patient access scheme (PAS). 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 hypoxia-inducible factor 2α (HIF-2α) inhibitor, belzutifan, blocks the action of HIF-2α.

Belzutifan has shown to be an effective treatment for Von Hippel-Lindau disease, 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 belzutifan was associated with a response rate of 64% in patients with RCC.

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.

Belzutifan is undergoing appraisal for use by NHS England for people with VHL disease. The outcome of this appraisal should be known early next year.

Action Kidney Cancer would like to thank those patients and clinicians who contributed to the belzutifan SMC 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 Medicines Advice on the SMC website here