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We are delighted to announce that thehas approved the use of the -inducible factor-2 alpha (HIF-2α) inhibitor, belzutifan, for the treatment of people with von Hippel-Lindau (VHL) 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 acalled the von Hippel-Lindau (VHL) , resulting in high levels of a protein called -inducible factor, or HIF-2α. This results in a number of changes in the and their surrounding environment that favour growth. The novel -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 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 is managed with surgery; tumours less than 3 cm in diameter are removed to limit the risk of spread of the and to avoid a decline in kidney function that results from multiple tumours in the kidney.. It results in
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. There is an ongoing phase 3 to compare belzutifan to in heavily pretreated patients and a combination with as a for advanced 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!