The National Medical Products Administration has approved Merck's (MRK, Financial) Belzutifan tablets for the treatment of von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, and pancreatic neuroendocrine tumors (pNET).
VHL is a rare genetic disorder affecting approximately 1 in 36,000 individuals. Patients with VHL are at risk of developing benign vascular tumors and certain cancers, including renal cell carcinoma.
Belzutifan is a small molecule inhibitor targeting hypoxia-inducible factor 2α (HIF-2α). It works by reducing the transcription and expression of HIF-2α target genes, which are associated with cell proliferation, angiogenesis, and tumor growth.
