MSD medicine accepted for use in Scotland

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MSD’s WELIREG^® (belzutifan) has been accepted for use within NHS Scotland in adults who have von Hippel-Lindau (VHL) disease.

This decision by the Scottish Medicines Consortium (SMC) relates to these patients who require therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNETs), and for whom localised procedures are unsuitable or undesirable.

VHL disease is a rare disorder that causes benign and malignant tumours and cysts to develop on different areas of the body, MSD stated.

Belzutifan

Belzutifan is an oral, novel hypoxia-inducible factor 2 alpha (HIF-2α) inhibitor. Its mechanism of action is based on research on HIF pathways and VHL genetics, for which, in 2019, a Nobel Prize was awarded, MSD highlighted.

The treatment was approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA) in May 2022. Its authorisation made the UK the first country in Europe with a license for this treatment. This occurred after belzutifan was the first product to receive an MHRA “innovation passport”, through the Innovation Licensing and Access Pathway (ILAP), the MHRA announced in 2021.

Clinical data supporting SMC acceptance

The recommendation was based on data from the Phase II LITESPARK-004 study, which enrolled 61 patients diagnosed with the rare hereditary disease. Patients received 120mg of belzutifan once per day. Belzutifan demonstrated clinically meaningful antitumour activity with overall response rates of at least 64 percent, 44 percent and 91 percent in RCC, CNS and pNETs, respectively.

As a result of the SMC’s acceptance, certain qualifying VHL patients in Scotland “will be the first in Europe to access belzutifan, fulfilling a huge unmet need for these VHL patients…” commented Graham Lovitt, Chair of VHL UK/Ireland.

“The decision [by the SMC] provides a welcome option for patients, reducing the need for and impact of numerous surgeries. We are committed to supporting the National Institute for Health and Care Excellence (NICE) with their ongoing appraisal to ensure that patients from the rest of the UK are able to access this treatment option,” David Long, Head of Oncology at MSD UK stated.