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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 12
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, in patients with advanced renal cell carcinoma who have received anti-PD-1 or PD-L1 therapy and VEGF inhibitor, and in adult and pediatric patients 12 years and older with pheochromocytoma or paraganglioma (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 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...
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 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...