Summary

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• Welireg (belzutifan) is indicated for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.
• This summary is based on the review of one randomized controlled trial(s). ^[1]
• For Renal Cell Carcinoma (RCC) associated with VHL disease, the objective response rate (ORR) was 49% (95% CI: 36 to 62) following a median follow-up of 21.8 months.
• For non-RCC neoplasms associated with VHL disease, the response rates were 77% for pancreatic lesions (47 out of 61 patients), 30% for central nervous system hemangioblastomas (15 out of 50 patients), and 100% improvement in all 16 evaluable eyes in patients with retinal hemangioblastomas.
• The most common adverse events reported were anemia (90% of patients) and fatigue (66% of patients).
• Serious adverse events included one treatment discontinuation due to grade 1 dizziness and one death attributed to acute toxic effects of fentanyl, not directly linked to the drug. Four additional patients voluntarily discontinued treatment, with one discontinuation due to disease progression.
• There is no population types or subgroups information available in the reviewed studies.