The National Health Service (NHS) England has announced the availability of MSD UK’s belzutifan, a take-at-home pill for patients with von Hippel-Lindau (VHL) disease.
The move builds on a positive recommendation from the UK National Institute for Health and Care Excellence (Nice).
Belzutifan is the first therapy to receive approval as a non-surgical treatment option for this life-threatening genetic condition.
The pill has shown promising results in clinical trials, with 95% of patients experiencing no tumour growth over two years and 56% reporting a reduction in tumour size.
Belzutifan, which is administered orally three times a day, simultaneously targets three different types of tumours.
It operates by hindering a protein that deprives cells of oxygen, a process that can cause normal cells to become tumorous. By stopping this protein, belzutifan prevents the growth of new tumours.
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As NHS England enters a commercial agreement with MSD UK for belzutifan, around 100 patients are expected to benefit from the therapy in the first year, and 50 annually thereafter.
The drug will be made available in the country through NHS England’s Cancer Drugs Fund.
NHS cancer national clinical director professor Peter Johnson stated: “This inherited illness is a shadow which hangs over entire families, who live with the fear of tumours growing and needing major surgery on many occasions.
“This new drug, taken as a tablet, is the first of its kind for this terrible condition, and patients with von Hippel-Lindau syndrome now have a different treatment option, which they can take in the comfort of their homes if they need it.
“It promises to improve the quality of life not just for those facing this disorder, but their families and carers too, and is a huge step forward in the treatment of this disease.”
