Therapy Detail

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Therapy Name Belzutifan
Synonyms
Therapy Description

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).

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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).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
VHL mutant islet cell tumor sensitive Belzutifan Guideline Actionable Welireg (belzutifan) is included in guidelines for patients with well-differentiated Grade 1/2 progressive pancreatic neuroendocrine tumors harboring germline VHL mutations (NCCN.org). detail...
VHL inact mut hemangioblastoma sensitive Belzutifan Guideline Actionable Welireg (belzutifan) is included in guidelines for patients with Von Hippel-Lindau disease-associated CNS hemangioblastoma that do not require immediate surgery (NCCN.org). 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 of 44% (22/61, 4 complete responses, 18 partial responses) in patients with Von Hippel-Lindau disease-associated CNS hemangioblastoma harboring germline VHL mutations, with a disease control rate of 90%, a median time to response of 5.4 months, and median progression-free survival not reached (J Clin Oncol 41, 2023 (suppl 16; abstr 2008); NCT03401788). detail... detail...
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 retinal hemangioblastoma sensitive Belzutifan Phase II Actionable In a Phase II trial, Welireg (belzutifan) treatment was safe and resulted in an overall response rate of 32% (16/50, 1 complete response, 15 partial responses) in patients with Von Hippel-Lindau disease-associated CNS hemangioblastoma harboring germline VHL mutations, 69% (11/16) of patients with retinal hemangioblastoma experienced improvement (J Clin Oncol 39, no. 15_suppl (May 20, 2021) 4555-4555; NCT03401788). 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...
VHL inact mut renal cell carcinoma sensitive Belzutifan Guideline Actionable Welireg (belzutifan) is included in guidelines for patients with Von Hippel-Lindau disease-associated renal cell carcinoma that do not require immediate surgery (NCCN.org). detail...

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT03401788 Phase II Belzutifan A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004) Active, not recruiting USA | GBR | FRA 1
NCT04489771 Phase II Belzutifan A Study of MK- 6482 in Participants With Advanced Renal Cell Carcinoma (MK-6482-013) Active, not recruiting USA | GBR | BEL | AUS 5
NCT02974738 Phase I Belzutifan A Trial of PT2977 Tablets In Patients With Advanced Solid Tumors Active, not recruiting 0
NCT05468697 Phase Ib/II Belzutifan + Palbociclib Belzutifan A Study of Belzutifan (MK-6482) in Combination With Palbociclib Versus Belzutifan Monotherapy in Participants With Advanced Renal Cell Carcinoma (MK-6482-024) Recruiting USA | AUS 1
NCT04924075 Phase II Belzutifan Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL) or Pancreatic Neuroendocrine Tumor (pNET) (MK-6482-015) Recruiting USA | ITA | GBR | FRA | ESP | DEU | CAN | AUS 10
NCT04846920 Phase I Belzutifan A Study of Belzutifan (MK-6482) in Participants With Advanced Clear Cell Renal Cell Carcinoma (MK-6482-018) Active, not recruiting USA 0
NCT04195750 Phase III Belzutifan Everolimus A Study of MK-6482 Versus Everolimus in Participants With Advanced Renal Cell Carcinoma (MK-6482-005) Active, not recruiting USA | ITA | GBR | FRA | ESP | DEU | CAN 16


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