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 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 statement is based on a regulatory approval from the European Medicines Agency:
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.