Summary

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• Belzutifan (Welireg) is indicated for the treatment of adult patients with von Hippel-Lindau disease who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors not requiring immediate surgery. The drug targets and inhibits hypoxia-inducible factor 2α, which becomes constitutively active due to VHL gene inactivation.
• A study from a randomized controlled trial was reviewed to gather information about Welireg's safety and effectiveness. This phase 2 open-label single-group trial focused on assessing its efficacy and safety specifically in treating renal cell carcinoma in patients with von Hippel-Lindau disease.
• In terms of effectiveness, Welireg achieved an objective response rate of 49% after a median follow-up of 21.8 months among patients with RCC. Significant activity was also noted against other VHL-related neoplasms; pancreatic lesions saw a response rate of 77%, CNS hemangioblastomas had a response rate of 30%, while all evaluated retinal hemangioblastomas showed improvement.
• Regarding the safety profile, the most common adverse events were anemia, affecting up to 90% of participants, and fatigue seen in around two-thirds (66%) of participants. These adverse events were mostly low-grade, indicating manageable side effects during the course of treatment.
• Seven outpatients discontinued treatment due to various reasons, including voluntary discontinuation and grade one dizziness as an adverse event related directly to the drug use itself, along with disease progression unrelated directly to usage such as acute toxic effect caused by fentanyl overdose, which ultimately led to death but was not linked directly to Welireg usage, highlighting relatively favorable adherence concerning overall health status during their respective courses of treatment.
• The trial focused on patients with von Hippel-Lindau disease who require therapy for associated RCC, CNS hemangioblastomas, or pNET not requiring immediate surgery. This indicates the potential utility of Welireg across a broad spectrum of VHL disease manifestations due to its high response rate in pancreatic lesions and improvement in retinal hemangioblastomas.