Belzutifan: First Approval

Belzutifan (Welireg (TM)) is an oral small molecule inhibitor of hypoxia-inducible factor (HIF)-2 alpha being developed by Peloton Therapeutics for the treatment of solid tumours, including renal cell carcinoma (RCC) with clear cell histology (ccRCC) and von Hippel-Lindau (VHL) disease-associated RCC. In August 2021, belzutifan received its first approval in the USA for the treatment of patients with VHL disease who require therapy for associated RCC, central nervous system (CNS) haemangioblastomas or pancreatic neuroendocrine tumours (pNET), not requiring immediate surgery. Clinical studies of belzutifan (as monotherapy or combination therapy) in other indications, including ccRCC, pNET and phaeochromocytoma/paraganglioma, are also underway in various countries. This article summarizes the milestones in the development of belzutifan leading to this first approval for certain VHL disease-associated tumours.