13 August 2021 - Today the FDA approved belzutifan (Welireg, Merck), a hypoxia-inducible factor inhibitor for adult patients with von Hippel-Lindau disease who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumours, not requiring immediate surgery.
Belzutifan was investigated in the ongoing Study 004, an open-label clinical trial in 61 patients with von Hippel-Lindau disease associated renal cell carcinoma diagnosed based on a von Hippel-Lindau disease germ-line alteration and with at least one measurable solid tumour localised to the kidney.