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.
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.
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.
Shares Hereditary pheochromocytoma and paraganglioma (SDHx, VHL, RET, NF1, MAX and TMEM127), New England Journal of Medicine.
Shares Hereditary pheochromocytoma and paraganglioma (SDHx, VHL, RET, NF1, MAX and TMEM127), Belzutifan.
Shares Hereditary pheochromocytoma and paraganglioma (SDHx, VHL, RET, NF1, MAX and TMEM127), Belzutifan.
Shares Hereditary pheochromocytoma and paraganglioma (SDHx, VHL, RET, NF1, MAX and TMEM127), Belzutifan.
Shares Hereditary pheochromocytoma and paraganglioma (SDHx, VHL, RET, NF1, MAX and TMEM127), Belzutifan.