Belzutifan
Orphan DrugFDA Approved
Description
Belzutifan is a hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor approved for the treatment of von Hippel-Lindau disease-associated tumors. It works by blocking HIF-2α, which is constitutively activated in VHL disease, leading to tumor growth. The drug has demonstrated efficacy in reducing the size of renal cell carcinomas, central nervous system hemangioblastomas, and pancreatic neuroendocrine tumors in VHL patients.
Indications & Therapeutic Use
von Hippel-Lindau disease-associated renal cell carcinoma, central nervous system hemangioblastomas, pancreatic neuroendocrine tumors
Linked Diseases:
von Hippel-Lindau disease
D006623
Global Availability (5 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Belzutifan
| Generic Name | Belzutifan |
| Brands | 1 brand available |
| Active Ingredient | Belzutifan |
| Drug Class | von Hippel-Lindau disease-associated renal cell carcinoma |
| Manufacturer | Merck & Co. |
| Dosage Forms | Oral tablet, 40mg |
| Medical Code | L01EX |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT03401788 |
| Countries | 5 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes