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 (MK-6482), blocks the action of HIF-2α.
Belzutifan has shown promise to treat a condition called 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, including kidney cancer. 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.
Belzutifan will become the first drug to benefit from a new fast-track approval process at the UK Medicines and Healthcare products Regulatory Agency (MHRA) aimed at speeding up patient access to breakthrough medicines. This process was first used for coronavirus vaccines and is intended to dramatically cut the time taken to license new drugs in the UK.
The MHRA announced the use of an “innovation passport” for belzutifan, enabling a decision to be made within 150 days after submission of trial data. The MHRA hopes that the new “innovative licensing and access pathway” will give the UK an edge over other medicines regulators and potentially persuade more pharmaceutical companies to develop their medicines in the UK.