Belzutifan for renal cell carcinoma and other tumours in von Hippel-Lindau disease
The oral HIF-2 alpha inhibitor belzutifan shrank kidney cancers in about half of people with von Hippel-Lindau disease and also shrank their brain and spinal haemangioblastomas and pancreatic tumours, letting many avoid repeated operations.
Overview
Phase 2 study of 61 patients with von Hippel-Lindau disease and at least one measurable renal cell carcinoma not needing immediate surgery, treated with belzutifan 120 mg daily.
Objective response in renal tumours was 49 percent, with responses in 30 percent of central nervous system haemangioblastomas and 77 percent of pancreatic neuroendocrine tumours; anaemia and hypoxia were the main side effects.
- Renal cell carcinoma objective response 49 percent.
- Central nervous system haemangioblastoma response 30 percent; pancreatic neuroendocrine tumour response 77 percent.
Belzutifan is the first systemic therapy for von Hippel-Lindau disease, approved for renal, central nervous system and pancreatic tumours not needing immediate surgery, changing a lifetime of surveillance and surgery for many patients.
- Single-arm study; long-term durability and the effect on survival are unknown.
- Anaemia in most patients, hypoxia in a minority; teratogenic.