Overview
Belzutifan is an inhibitor of hypoxia-inducible factor 2α (HIF-2α) used in the treatment of von Hippel-Lindau (VHL) disease-associated cancers. 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. Initially considered to be undruggable, a binding pocket was eventually discovered in the HIF-2α molecule which allowed for compounds to bind and inhibit these proteins. This discovery led to the initial development of belzutifan (at the time called PT2977), which was further developed by a spin-off company named Peloton Pharmaceuticals (which itself was eventually acquired by Merck in 2019). Belzutifan inhibits the complexation of HIF-2α with another transcription factor, HIF-1β, a necessary step in its activation - by preventing the formation of this complex, belzutifan can slow or stop the growth of VHL-associated tumors. Belzutifan received FDA approval for the treatment of select VHL-associated cancers on August 13, 2021.
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 not require immediate surgery.
Associated Conditions
- Hemangioblastoma
- Pancreatic Neuroendocrine Cancer
- Renal Cell Carcinoma (RCC)
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/07/03 | Not Applicable | Not yet recruiting | |||
2025/06/17 | Phase 2 | Not yet recruiting | |||
2025/04/01 | Phase 1 | Completed | |||
2024/11/06 | Phase 2 | Recruiting | |||
2024/05/24 | Phase 2 | Recruiting | |||
2024/01/31 | Phase 1 | Recruiting | |||
2023/06/12 | Phase 3 | Active, not recruiting | |||
2022/07/21 | Phase 1 | Active, not recruiting | |||
2022/02/15 | Phase 3 | Active, not recruiting | |||
2021/09/01 | Phase 1 | Active, not recruiting |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Merck Sharp & Dohme LLC | 0006-5331 | ORAL | 40 mg in 1 1 | 12/14/2023 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 2/12/2025 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| WELIREG® FILM-COATED TABLET 40MG | SIN17233P | TABLET, FILM COATED | 40.00mg | 5/9/2025 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| WELIREG belzutifan 40 mg film-coated tablet blister pack | 482705 | Medicine | A | 6/25/2025 | |
| WELIREG belzutifan 40 mg film-coated tablet bottle | 355338 | Medicine | A | 12/22/2022 |
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| WELIREG | Merck Canada Inc | 02528908 | Tablet - Oral | 40 MG | 9/1/2022 |
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| WELIREG 40 MG COMPRIMIDOS RECUBIERTOS CON PELICULA | 1241893002 | COMPRIMIDO RECUBIERTO CON PELÍCULA | Diagnóstico Hospitalario | Not Commercialized |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
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