following a full submission assessed under the orphan medicine process:
belzutifan (Welireg®) is accepted for use within NHSScotland.
Indication under review: treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable.
In a single-arm, phase II study, belzutifan was associated with overall response rates of at least 64%, 44% and 91% in RCC, CNS and pNET, respectively.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Download detailed advice350KB (PDF)
Medicine details
- Medicine name:
- belzutifan (Welireg)
- SMC ID:
- SMC2587
- Indication:
Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable.
- Pharmaceutical company
- Merck Sharp & Dohme Ltd
- BNF chapter
- Malignant disease and immunosuppression
- Submission type
- Full
- Status
- Accepted
- Date advice published
- 09 October 2023