NHS patients with a ‘life-threatening’ tumour-causing disease will now benefit from a first-ever drug treatment.
The National Institute for Health and Care Excellence (NICE) has recommended belzutifan, sold under the brand name Welireg, as a treatment for adults with von Hippel-Lindau disease (VHL), a rare genetic disorder.
Helen Knight, director of medicines evaluation for NICE, said that VHL severely affects the quality of life for patients and their families. ‘Currently, there are no licensed medicines targeting its underlying causes, with surgery being the main treatment option for removing tumours and cysts. Belzutifan is a new treatment that has the potential to prevent loss of organ function from repeated tumours and surgeries and [to] improve people’s quality of life at a price that is good value for the taxpayer.’
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Around 800 people in England live with VHL disease, which is a genetic condition that causes an abundance in proteins that can starve cells of oxygen, which leads normal cells to transform into tumour cells.
People with VHL usually need multiple surgeries throughout their lives, and because of the location of tumours in the brain, spinal cord, eyes, pancreas and kidneys, procedures to remove tumours are often high-risk, and can have life-changing consequences, including paralysis, loss of vision, diabetes, or the need for life-long dialysis.
The new oral drug stops the excess proteins from starving cells of oxygen and prevents the growth of tumours. In NHS clinical trials, 95% of patients did not experience any growth in their tumours in two years of taking belzutifan, and 56% of patients’ tumours shrunk.
The chair of VHL UK/Ireland, Graham Lovitt, welcomed NICE’s recommendation, calling it a ‘significant milestone in the fight against this rare disease’.
‘This decision brings hope to patients and their families, offering them an alternative treatment option, where others become too high risk.’