Regulatory approval

Published by the Food and Drug Administration.

The U.S. Food and Drug Administration (FDA) granted approval to belzutifan for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNETs), not requiring immediate surgery. 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 a hypoxia-inducible factor inhibitor indicated for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNETs), not requiring immediate surgery.

Citation

Merck Sharp & Dohme LLC. Welireg (belzutifan) [package insert]. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/215383s012lbl.pdf. Revised May 2025. Accessed May 6, 2026.

Therapeutic response

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

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