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Title Welireg (belzutifan) FDA Drug Label
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URL https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215383
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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Belzutifan Welireg PT2977|MK-6482|PT-2977 HIF2A Inhibitor 5 Welireg (belzutifan) is a hypoxia inducible factor-2alpha (EPAS1, HIF2A) inhibitor that suppresses expression of HIF2A target genes, resulting in tumor regression (Mol Cancer Ther 2018,17(1 Suppl):Abstract nr B140, PMID: 31282155). Welireg (belzutifan) is FDA approved for use in patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors that require therapy (FDA.gov).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
VHL inact mut pancreatic endocrine carcinoma sensitive Belzutifan FDA approved Actionable In a Phase II trial that supported FDA approval, Welireg (belzutifan) treatment was safe and resulted in an overall response rate of 80% (16/20, 1 complete response, 15 partial responses) in patients with Von Hippel-Lindau disease-associated pancreatic neuroendocrine tumors harboring germline VHL mutations (J Clin Oncol 39, no. 15_suppl (May 20, 2021) 4555-4555; NCT03401788). detail... detail...
VHL inact mut renal cell carcinoma sensitive Belzutifan FDA approved Actionable In a Phase II trial that supported FDA approval, Welireg (belzutifan) treatment was safe and resulted in a confirmed partial response (PR) in 36% (22/61) and unconfirmed PR in 11% (7/61) of patients with Von Hippel-Lindau disease-associated localized renal cell carcinoma harboring germline VHL mutations, median duration of response was not reached in responders (J Clin Oncol 39, no. 15_suppl (May 20, 2021) 4555-4555; NCT03401788). detail... detail...