Abstract
Transcription factor HIF-2α is an established oncogenic driver, and its activation in VHL disease is caused by germline alterations in the VHL gene. Belzutifan is a first-in-class HIF-2α inhibitor approved for patients (pts) with VHL disease who require therapy for associated renal cell carcinoma (RCC), CNS hemangioblastomas, or pancreatic neuroendocrine tumors (pNET) not requiring immediate surgery. Results with more than 3 years of follow-up from the phase 2 LITESPARK-004 study (NCT03401788) are presented.
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