Belzutifan is an inhibitor of hypoxia-inducible factor 2α used as an antineoplastic in the treatment of certain cancers associated with von Hippel-Lindau (VHL) disease. The HIF-2α protein was first identified in the 1990s by researchers at UT Southwestern Medical Center as a key player in the growth of certain cancers.
- Mechanism
- Curator reviewed · 3 references
Hypoxia-inducible factor 2α (HIF-2α) is a transcription factor which aids in oxygen sensing by regulating genes that promote adaptation to hypoxia. In healthy patients, when oxygen levels are normal, HIF-2α is broken down via ubiquitin-proteasomal degradation by von-Hippel Lindau (VHL) proteins. In the presence of hypoxia, HIF-2α translocates into cell nuclei and forms a transcriptional complex with hypoxia-inducible factor 1β (HIF-1β) - this complex then induces the expression of downstream genes associated with cellular proliferation and angiogenesis.
Patients with von-Hippel Lindau (VHL) disease lack functional VHL proteins, leading to an accumulation of HIF-2α, and this accumulation is what drives the growth of VHL-associated tumors. Belzutifan is an inhibitor of HIF-2α that prevents its complexation with HIF-1β in conditions of hypoxia or impaired VHL protein function, thereby reducing the expression of HIF-2α target genes and slowing/stopping the growth of VHL-associated tumors.
- Primary indication
- Belzutifan is indicated for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), who do...Curator reviewed · 8 structured indications
- Formula / weight
- C17H12F3NO4S · 383.34 g/mol (avg)
- First approval
- Canada, 2022 · United States, 2021 · European Union, 2025
- Code names
- MK-6482 · PT-2977 · WHO 11196
- Brand names
Resolves to
LOMMPXLFBTZENJ-ZACQAIPSSA-NSMILESCS(=O)(=O)C1=CC=C(OC2=CC(=CC(F)=C2)C#N)C2=C1[C@H](O)[C@H](F)[C@@H]2FWhat you can answer from here — as of September 11, 2026
Which drugs share a target with Belzutifan, and which of those have an active Phase 3 trial?
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