Therapeutic Response

VHL pathogenic variants status confers therapeutic sensitivity to Belzutifan in patients with Central Nervous System Hemangioblastoma.

Statements

Source and description
Welireg (belzutifan) [product information]. EMA. 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.
Welireg (belzutifan) [package insert]. FDA. 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.