NHS England provides MSD belzutifan for von Hippel-Lindau disease

The National Health Service (NHS) England has announced the introduction of belzutifan, a new oral medication developed by MSD UK for patients with von Hippel-Lindau (VHL) disease. This initiative follows a favorable recommendation from the UK National Institute for Health and Care Excellence (NICE).

Belzutifan is noteworthy as the first approved non-surgical treatment for VHL, a severe genetic disorder. Clinical trials have demonstrated its effectiveness, with 95% of participants experiencing no tumour growth over a two-year period and 56% seeing a reduction in tumour size. The drug is designed to be taken at home, three times daily, and targets three different tumour types simultaneously.

The mechanism of belzutifan involves inhibiting a specific protein that deprives cells of oxygen, causing normal cells to turn tumorous. By blocking this protein, belzutifan effectively halts the formation of new tumours. 

NHS England has entered into a commercial agreement with MSD UK to supply belzutifan. Initially, around 100 patients are expected to benefit from this treatment in its first year, with an anticipated 50 patients annually thereafter. The drug will be provided through NHS England’s Cancer Drugs Fund.

Professor Peter Johnson, the NHS cancer national clinical director, highlighted the significant impact of this drug on patients and their families. He noted that VHL is an inherited condition that casts a long shadow over affected families, who live with the constant threat of tumour growth and the need for repeated major surgeries. Johnson emphasized that this new treatment, administered as a tablet, offers a non-invasive alternative that patients can take at home, significantly improving their quality of life.

This development not only represents a major advancement for those suffering from VHL but also offers hope and relief to their families and caregivers, marking a significant step forward in the management of this debilitating disease.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!