New treatment for rare disease that causes brain tumours

Nicola Gale

2 min read

The first treatment for a rare condition which causes brain tumours has been announced today.

The drug belzutifan is now available on the NHS as treatment for Von Hippel-Lindau (VHL) disease, a rare condition which causes cysts and tumours in different parts of the body, including the brain and spinal cord.

The National Institute for Health and Care Excellence (NICE) has recommended belzutifan for some adults with VHL disease who need treatment for VHL-associated central nervous system, kidney or pancreatic tumours.

Evidence shows belzutifan can reduce tumour size or prevent them from getting bigger and prevent the loss of organ function that people experience from repeated tumours and surgeries. 

Helen Knight, Director of Medicines Evaluation at NICE, said: “This condition severely affects the quality of life of people who are living with it, as well as their families and carers.  

“There are no medicines that are currently recommended by NICE that address the underlying causes of this condition and surgery is the main treatment option for removing tumours and cysts. 

“This is a new treatment that has the potential to prevent loss of organ function from repeated tumours and surgeries and improve people’s quality of life at a price that is good value for the taxpayer. We’re therefore pleased to be able to recommend it today while more evidence is collected.”

Belzutifan will be available under managed access on the NHS. Managed access gives patients faster access to promising new treatments that might not be recommended because of uncertainties about their clinical or cost effectiveness. During managed access, more evidence is collected to address any uncertainties about a treatment and NICE then uses this evidence to assess whether the treatment should be made available for routine use on the NHS.

Dr Karen Noble, Our Director of Research, Policy and Innovation, said: “There are approximately 600 people living with VHL in England and seeing a new treatment that may increase the time between surgeries and increase quality of life is welcomed. We will be keeping a close eye on belzutifan while it’s assessed by NICE and hope to see it approved for routine use”.