MSD’s – known as Merck & Co in the US and Canada – Welireg (belzutifan) has been recommended with managed access by the National Institute for Health and Care Excellence (NICE) to treat a rare genetic disease that causes tumours in major organs.
The take-at-home tablet will be available on the NHS in England and Wales to treat von Hippel-Lindau (VHL) disease in adults who require therapy for VHL-associated renal cell carcinomas, central nervous system haemangioblastomas or pancreatic neuroendocrine tumours, and for whom localised procedures are unsuitable or undesirable.
Affecting approximately one in every 70,000 people, VHL disease occurs when a mutation in the VHL gene causes cells to grow abnormally. This can lead to cysts or tumours developing in different parts of the body, including the kidneys, brain and pancreas that can impair function or become cancerous.
Until now, the only treatments available have been invasive surgery or radiotherapy, with patients usually requiring multiple surgeries throughout their lives.
Benson Fayehun, head of oncology at MSD UK said: “VHL disease is a complex and devastating genetic condition, with patients often acting as carers for their family members, who may also have a VHL diagnosis. Until now, these patients have had few treatment options, typically limited to invasive procedures.
“This decision provides a welcome option for patients, reducing the need for and impact of numerous surgeries.”
Around 100 people could benefit from Welireg in the first year and approximately 50 people per year thereafter, according to NHS England.
NICE’s final draft guidance on the drug, which was enabled by collaboration between NHS England and MSD to provide access through the Cancer Drugs Fund, was supported by clinical trial results showing that 95% of patients did not experience any growth in their tumours in two years of taking the treatment and 56% of patients’ tumours shrunk.
However, the agency noted that there are “some uncertainties” and that more evidence is needed before Welireg can be considered for routine use on the NHS, so it has been recommended for use with further data collection.
Helen Knight, director of medicines evaluation at NICE, 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.”
