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Molecular Profile VHL inact mut
Therapy Belzutifan
Indication/Tumor Type pancreatic endocrine carcinoma
Response Type sensitive

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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 (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...