Regulatory approval

Published by the European Medicines Agency.

The European Medicines Agency (EMA) has authorized belzutifan as a monotherapy for the treatment of adult patients with von Hippel-Lindau disease who require therapy for associated, localized renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable. This indication is based on LITESPARK-004, an open-label phase 2 clinical study in 61 patients with VHL disease who had at least one measurable solid tumor localized to the kidney and who did not require immediate surgery. Patients could also have other VHL disease-associated tumors such as CNS hemangioblastomas and pNETs. VHL-associated disease was based on the presence of a VHL germline alteration.

This is written in the approval document as:

Welireg is indicated as monotherapy for the treatment of adult patients with von Hippel-Lindau disease who require therapy for associated, localised renal cell carcinoma (RCC), central nervous system (CNS) haemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable.

Citation

Merck Sharp & Dohme B.V. Welireg (belzutifan) [product information]. European Medicines Agency website. https://www.ema.europa.eu/en/documents/product-information/welireg-epar-product-information_en.pdf.pdf. Revised January 2026. Accessed May 5, 2026.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Organization(s)	Biomarker(s)	Cancer type	Therapy(ies)
EMA (1)	VHL pathogenic variants	Renal Cell Carcinoma	Belzutifan
EMA (1)	VHL pathogenic variants	Central Nervous System Hemangioblastoma	Belzutifan
EMA (1)	VHL pathogenic variants	Pancreatic Neuroendocrine Tumor	Belzutifan