Belzutifan Approved by FDA for von Hippel-Lindau Disease Associated With Kidney Cancer
Posted: Wednesday, August 18, 2021
On August 13, the U.S. Food and Drug Administration (FDA) approved the hypoxia-inducible factor inhibitor belzutifan (Welireg) for the treatment of adults with von Hippel-Lindau disease (VHL) who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors that do not require immediate surgery. The recommended dosage is 120 mg administered orally once daily with or without food.
The FDA approval was based on the results of the ongoing, open-label Study 004, which investigated the efficacy of belzutifan on VHL-associated renal cell carcinoma based on a VHL germline alteration and with at least one measurable solid tumor localized to the kidneys. All patients with VHL-associated renal cell carcinoma with a response were followed for a minimum of 18 months from the start of treatment. Patients were administered 120 mg of belzutifan once daily until disease progression or unacceptable toxicity.
There was a 49% overall response rate with belzutifan in patients with VHL-associated renal cell carcinoma. Although the median duration of response was not reached, about 56% of responders had a duration of response of 12 months or more and a time to response of 8 months.
The most common adverse reactions observed in 20% or more of patients who received belzutifan included decreased hemoglobin levels, anemia, fatigue, increased creatinine levels, headache, dizziness, increased glucose, and nausea. Anemia occurred in 90% of patients, and 7% had grade 3 anemia. Hypoxia related to the use of belzutifan occurred in 1.6% of patients. Belzutifan may also render some hormonal contraceptives ineffective, and belzutifan exposure during pregnancy may cause embryofetal harm.
For more prescribing information on belzutifan, visit accessdata.fda.gov.