Therapeutic Response Statement (Sensitivity)
This therapeutic response statement supports the relationship that VHL pathogenic variants status confers therapeutic sensitivity to Belzutifan in patients with Pancreatic Neuroendocrine Tumor.
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 statement is based on a regulatory approval from the Food and Drug Administration:
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.