Belzutifan (2) – Welireg®

• Clinical trials
  • The ongoing, open-label, single-arm LITESPARK 004 trial is the pivotal Phase II trial on the basis of which belzutifan was granted conditional approval.
  • The Von Hippel-Lindau Natural History Study is a retrospective, non-interventional study based on data from the National Cancer Institute Urologic Oncology Branch Von Hippel-Lindau Hereditary Database.

Adults with renal cell carcinoma (RCC) associated with von Hippel-Lindau syndrome, central nervous system (CNS) haemangioblastoma or pancreatic neuroendocrine tumour (pNET) associated with von Hippel-Lindau syndrome, for whom local therapies are not an option and who require treatment

• An additional benefit is not proven.
• Overall, the data presented are not sufficient to demonstrate any additional benefit of belzutifan compared with the appropriate comparator therapy; therefore, there is no additional benefit of belzutifan for the treatment of adults with renal cell carcinoma (RCC), central nervous system (CNS) haemangioblastoma or pancreatic neuroendocrine tumour (pNET), for whom local therapies are not an option and who require treatment, is not proven.
• Conclusion
  • Overall, the data presented are not sufficient to demonstrate any additional benefit of belzutifan compared with the appropriate comparator therapy; therefore, no additional benefit of belzutifan has been demonstrated for the treatment of adults with renal cell carcinoma associated with von Hippel–Lindau syndrome (RCC), central nervous system (CNS) haemangioblastoma or pancreatic neuroendocrine tumour (pNET), for whom local therapies are not an option and who require treatment, is not proven.

Courtesy translation only, please refer to the German original.