Drug updated on 9/4/2024
Dosage Form | Tablet (oral; 40 mg) |
Drug Class | Hypoxia-inducible factor inhibitors |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- 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), not requiring immediate surgery.
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Summary
- Welireg (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), not requiring immediate surgery.
- This summary is based on the review of one randomized controlled trial(s). [1]
- For Renal Cell Carcinoma (RCC) associated with VHL disease, the objective response rate (ORR) was 49% (95% CI: 36 to 62) following a median follow-up of 21.8 months.
- For non-RCC neoplasms associated with VHL disease, the response rates were 77% for pancreatic lesions (47 out of 61 patients), 30% for central nervous system hemangioblastomas (15 out of 50 patients), and 100% improvement in all 16 evaluable eyes in patients with retinal hemangioblastomas.
- The most common adverse events reported were anemia (90% of patients) and fatigue (66% of patients).
- Serious adverse events included one treatment discontinuation due to grade 1 dizziness and one death attributed to acute toxic effects of fentanyl, not directly linked to the drug. Four additional patients voluntarily discontinued treatment, with one discontinuation due to disease progression.
- There is no population types or subgroups information available in the reviewed studies.
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 |