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Innovation Observatory > Reports > Drugs > Belzutifan for von Hippel-Lindau disease-associated clear cell renal cell carcinoma – first line

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Belzutifan for von Hippel-Lindau disease-associated clear cell renal cell carcinoma – first line

Drugs

Cancer and Palliative Care

January 2021


Belzutifan is in clinical development for the treatment of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC). VHL is a hereditary disease that results in the body producing an increased amount of the protein hypoxia inducible factor (HIF) – 2α. This leads to an increased risk of tumours developing in various parts of the body including the kidney. Renal cell carcinoma (RCC) is the most common type of kidney cancer. ccRCC is named as such because when this type of tumour is viewed under a microscope the cells appear clear. Currently patients with VHL disease have limited treatment options which focus on surgically removing tumours once they have developed.
Belzutifan is a first-in-class drug that is taken orally and works by selectively blocking the activity of the HIF-2α protein. HIF-2α plays a role in cancer cell survival, cell growth and blood vessel formation so by blocking this protein belzutifan is thought to slow down the worsening of the disease and improve symptoms. If licensed, belzutifan may offer an additional treatment option for patients with VHL disease-associated ccRCC.

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