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Ref Type
PMID
Authors
Title Welireg (belzutifan) FDA Drug Label
Journal Vol Issue Date Pages Journal Short Title
URL https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215383
Abstract Text

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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
Belzutifan Belzutifan 10 8
Drug Name Trade Name Synonyms Drug Classes Drug Description
Belzutifan Welireg PT2977|MK-6482|PT-2977 HIF2A Inhibitor 6 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, and in patients with advanced renal cell carcinoma who have received anti-PD-1 or PD-L1 therapy and VEGF inhibitor (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 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 an objective response in 49% (30/61, 30 partial responses) and stable disease in 49% of patients with Von Hippel-Lindau disease-associated localized renal cell carcinoma harboring germline VHL mutations, 94% of patients were progression-free at 24 months and median duration of response was not reached with a median follow-up of 21.8 months (PMID: 34818478; NCT03401788). 34818478 detail...
VHL inact mut pancreatic endocrine carcinoma sensitive Belzutifan FDA approved Actionable In a Phase II trial (LITESPARK-004) that supported FDA approval, Welireg (belzutifan) treatment was safe and resulted in an overall response rate (ORR) of 84% (51/61) in patients with Von Hippel-Lindau disease-associated pancreatic tumors harboring germline VHL mutations, ORR was 91% (20/22, 7 complete responses) in patients with pancreatic neuroendocrine tumors, with median duration of response and overall survival not reached at a median follow up of 37.8 months (PMID: 38393723; NCT03401788). 38393723 detail...
VHL inact mut hemangioblastoma sensitive Belzutifan FDA approved Actionable In a Phase II trial (LITESPARK-004) that supported FDA approval, Welireg (belzutifan) treatment was safe and resulted in an objective response rate (ORR) of 44% (22/50) in patients with Von Hippel-Lindau disease-associated CNS hemangioblastoma harboring germline VHL mutations, ORR was 76% (19/25) in those with measurable baseline lesions (PMID: 39284337; NCT03401788). 39284337 detail...