Drug updated on 3/28/2024
| Dosage Form | Tablet (oral; 40 mg) |
| Drug Class | Hypoxia-inducible factor inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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), not requiring immediate surgery.
Summary
- Belzutifan (Welireg) is a hypoxia-inducible factor 2α (HIF-2α) inhibitor used for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors not requiring immediate surgery.
- The document reviewed was a phase 2 open-label single-group trial that investigated the efficacy and safety of belzutifan in treating VHL-associated diseases; it's one among randomized controlled trials on this drug.
- In this study, belzutifan was administered orally at a dose of 120 mg daily to patients with renal cell carcinoma associated with VHL disease; after median follow-up period of approximately 22 months, an objective response rate - complete or partial - was observed in about half these patients.
- Besides its effectiveness against renal cell carcinomas, responses were also noted in cases involving non-renal neoplasms such as pancreatic lesions and central nervous system hemangioblastomas where improvement rates reached up to 77% and 30%, respectively.
- All evaluated eyes from patients suffering retinal hemangioblastomas showed signs of improvement post-treatment indicating potential benefits beyond primary indications mentioned earlier.
- While predominantly grade one and two adverse events like anemia and fatigue were reported by majority participants during the trial period leading seven out them discontinuing treatment due to various reasons including voluntary withdrawal, dizziness related side-effects etc., no severe complications directly attributable to Welireg administration surfaced making it relatively safe compared other drugs available for similar conditions currently on market.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Welireg (belzutifan) Prescribing Information. | 2022 | Merck & Co. Inc., Rahway, NJ |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Belzutifan for renal cell carcinoma in von Hippel–Lindau disease. | 61Subjects F: 48% M: 52% | 2021 | New England Journal of Medicine |
Sex Distribution:
F:48%
M:52%
61Subjects
Year:
2021
Source:New England Journal of Medicine
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| von Hippel-Lindau disease: Updated guideline for diagnosis and surveillance. | 2022 | European Journal of Medical Genetics |