FDA approves Welireg for von Hippel-Lindau disease-associated cancers
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The FDA approved belzutifan for treatment of cancers associated with von Hippel-Lindau disease.
The indication applies to use of the agent for adults with von Hippel-Lindau disease who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas or pancreatic neuroendocrine tumors that do not require immediate surgery.
Belzutifan (Welireg, Merck) is a hypoxia-inducible factor inhibitor.
The ongoing, open-label Study 004 is evaluating the agent for 61 patients with von Hippel-Lindau disease-associated renal cell carcinoma who have at least one measurable solid tumor localized to the kidney. Researchers also enrolled patients who had other von Hippel-Lindau disease-associated tumors, including central nervous system hemangioblastomas or pancreatic neuroendocrine tumors.
Patients received 120 mg belzutifan once daily. Treatment continued until disease progression or unacceptable toxicity.
Overall response rate assessed by an independent review committee served as the primary efficacy endpoint. Additional efficacy endpoints included time to response and duration of response.
Researchers reported an ORR of 49% (95% CI, 36-62) among patients with von Hippel-Lindau disease-associated renal cell carcinoma. Median time to response was 8 months. Median duration of response had not been reached; however, 56% of patients achieved response that lasted for at least 1 year.
Among 24 patients with measurable CNS hemangioblastomas, 63% responded to treatment and 73% of responses lasted at least 12 months. Among 12 patients with measurable pancreatic neuroendocrine tumors, 83% responded to treatment and 50% of responses lasted at least 12 months. Median duration of response in these subgroups had not been reached.
A majority (90%) of patients in Study 004 developed anemia (grade 3, 7%). Hypoxia occurred among 1.6% of patients.
Other adverse events reported among at least 20% of patients included decreased hemoglobin, anemia, fatigue, increased creatinine, headache, dizziness, increased glucose and nausea.
The FDA previously granted priority review to belzutifan for this indication.
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