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NICE has recommended belzutifan for some adults with Von Hippel-Lindau disease, in final draft guidance, which could benefit up to 100 people.

Von Hippel-Lindau disease (VHL) is a rare genetic condition caused by a mutation in the VHL gene. This gene produces a protein that controls cell growth, and a mutation in this gene can cause cells to grow abnormally. This can lead to cysts or tumours developing in different parts of the body, such as the kidneys, brain and pancreas that can impair function or become cancerous. 

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. 

VHL varies from person to person and although some people might only develop 1 or a few tumours in their whole life, others might have multiple tumours in different (or the same) organs. There are only about 600 adults in England living with the condition. 

NICE is looking at belzutifan for adults who need treatment for VHL-associated kidney, central nervous system or pancreatic tumours. 

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.

Helen Knight, director of medicines evaluation

The committee heard from patient experts who explained that for some people there are a wide variety of debilitating symptoms depending on where the tumours are growing in the body. These include constant pain, loss of balance and motor skills, loss of vision, breathlessness, coughing, headaches, confusion, severe nausea and fatigue.  

Evidence from a small study suggests that belzutifan reduces tumour size and gives people longer before their condition gets worse. However there are some uncertainties and more evidence is needed before it can be considered for routine use on the NHS, so it is recommended for use with further data collection; known as managed access. 

Managed access gives people 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.

Belzutifan for treating tumours associated with von Hippel-Lindau disease