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Molecular Profile | VHL inact mut |
Therapy | Belzutifan |
Indication/Tumor Type | pancreatic endocrine carcinoma |
Response Type | sensitive |
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 (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... |
PubMed Id | Reference Title | Details |
---|---|---|
Welireg (belzutifan) FDA Drug Label | Full reference... | |
(38393723) | Belzutifan for von Hippel-Lindau Disease: Pancreatic Lesion Population of the Phase 2 LITESPARK-004 Study. | Full reference... |