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 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 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 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 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
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 | NLD | ITA | GBR | FRA | ESP | DEU | CAN | AUS 9
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
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
NCT04489771 Phase II Belzutifan A Study of MK- 6482 in Participants With Advanced Renal Cell Carcinoma (MK-6482-013) Active, not recruiting USA | NLD | GBR | BEL | AUS 4
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
NCT02974738 Phase I Belzutifan A Trial of PT2977 Tablets In Patients With Advanced Solid Tumors Active, not recruiting 0
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


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