NICE Recommends MSD’s Welireg for Treating Rare Genetic Condition, Von Hippel-Lindau Disease

Welireg (belzutifan), developed by MSD (known as Merck & Co in the US and Canada), has received a recommendation from the National Institute for Health and Care Excellence (NICE) for managing Von Hippel-Lindau (VHL) disease. This rare genetic condition, affecting approximately one in every 70,000 people, causes abnormal tumour growth in vital organs.

The newly approved take-at-home tablet will soon be available on the NHS in England and Wales for adult patients suffering from VHL-associated renal cell carcinomas, central nervous system haemangioblastomas, or pancreatic neuroendocrine tumours, particularly for those who are unsuitable for or do not wish to undergo localized surgical interventions.

VHL Disease and Current Treatment Challenges

VHL disease is caused by a mutation in the VHL gene, which leads to abnormal cell growth. This growth can result in cysts or tumours in organs such as the kidneys, brain, and pancreas, which may compromise organ function or evolve into cancer. Until the approval of Welireg, patients primarily relied on invasive surgeries or radiotherapy, often requiring multiple procedures throughout their lifetimes to manage the condition.

Benson Fayehun, MSD UK’s head of oncology, emphasized the importance of this development: “VHL disease is a devastating genetic condition, and until now, patients have had few treatment options beyond invasive procedures. Welireg offers a much-needed alternative, reducing the burden of surgeries for patients.”

A New Hope for Patients

Clinical trials have shown promising results, with 95% of patients not experiencing tumour growth over two years, and 56% seeing tumour shrinkage. While NICE recognizes some uncertainties regarding long-term outcomes, the drug has been approved under managed access, allowing for further data collection while making it available to patients.

Helen Knight, director of medicines evaluation at NICE, stated, “This treatment has the potential to improve the quality of life for people affected by repeated tumours and surgeries. We are pleased to recommend it while continuing to collect additional evidence.”

Around 100 patients are expected to benefit from Welireg in its first year of availability, with around 50 patients per year thereafter.