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On August 13, 2021, the US FDA approved Welireg (belzutifan), a hypoxia-inducible factor inhibitor developed by Merck (MSD), for the treatment of von Hippel-Lindau (VHL) disease-related cancers, such as renal cell carcinoma (RCC), Central nervous system (CNS) hemangioblastoma or pancreatic neuroendocrine tumor (pNET), these patients do not need immediate surgical treatment
VHL is a rare genetic disease that can abnormally activate hypoxia-inducible factor (HIF-2α) in cancer patients
This approval is based on the results of an ongoing Phase 2 clinical trial
▲Belzutifan molecular structure (picture source: DMacks, Public domain, via Wikimedia Commons)
In terms of safety, the most common adverse reactions reported in patients in the belzutifan group were decreased hemoglobin, anemia, fatigue, increased creatinine, headache, dizziness, increased glucose, and nausea
Reference materials:
[1] FDA approves belzutifan for cancers associated with von Hippel-Lindau disease.