New treatment option for people with rare genetic condition

In News

Follow this topic

Bookmark

Record learning outcomes

In the final draft of guidance published on September 5, NICE has recommended belzutifan for some adults with Von Hippel-Lindau (VHL) disease.  The rare genetic condition is caused by a mutation in the VHL gene which produces a protein that controls cell growth. The mutation in this gene can cause cells to grown abnormally, leading to tumours developing in different parts of the body that can impair organ function or become cancerous. Belzutifan has been shown to reduce tumour size or prevent them from getting bigger. NICE is looking at belzutafin for adults who need treatment for VHL-associated kidney, central nervous system or pancreatic tumours.  

Helen Knight, director medicines evaluation, said: “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.”