NHS patients in England with von Hippel-Lindau (VHL) disease now have access to the first-ever drug specifically approved for their condition. Belzutifan, a groundbreaking oral medication, has been greenlit by the National Institute for Health and Care Excellence (NICE) and will be available through the NHS from September 5. The approval follows a successful commercial deal between NHS England and drug manufacturer MSD UK.
VHL disease is a rare, hereditary disorder characterized by the growth of multiple, malignant and benign tumors in major organs, including the brain, spine, eyes, pancreas, and kidneys. Approximately 800 people in England are affected by this condition, which often necessitates frequent and high-risk surgeries due to the location and nature of the tumors. Traditional treatments include invasive surgery and radiotherapy, which can have severe life-altering consequences, such as paralysis or loss of vision.
Belzutifan, administered as a pill taken three times daily, represents a significant advancement in the treatment of VHL disease. Clinical trials have demonstrated that 95% of patients experienced no tumor growth over two years of treatment, and 56% saw their tumors shrink. This new drug offers patients an option to avoid or delay high-risk surgeries, potentially improving their quality of life.
The availability of Belzutifan is expected to benefit around 100 patients in its first year, with approximately 50 additional patients annually thereafter. The drug will be accessible through NHS England’s Cancer Drugs Fund while further evidence on its effectiveness is gathered.
Ella Fox-Stillwell, a 26-year-old nurse with VHL, expressed her relief and hope for the future with this new treatment option. “This drug could change our lives,” she said, highlighting its potential to offer hope where traditional treatments fall short.
Professor Peter Johnson, NHS National Clinical Director for Cancer, praised the approval, noting, “This new drug is a crucial step forward for patients with VHL syndrome, offering them a more manageable treatment option that can be taken at home.”
Belzutifan works by inhibiting a protein that contributes to tumors growth by depriving cells of oxygen, a mechanism linked to the Nobel Prize-winning research on cancer development. This innovative therapy marks a pivotal moment in the fight against VHL disease, providing new hope for patients and their families.
Chair of VHL UK/Ireland, Graham Lovitt, welcomed the approval as a significant milestone, while Benson Fayehun, Head of Oncology at MSD UK, expressed satisfaction that patients will now have a viable alternative to invasive procedures.
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