FDA Grants Priority Review to HIF-2α Inhibitor for Renal Cell Carcinoma
Posted: Tuesday, March 16, 2021
On March 16, Merck & Co. announced that the U.S. Food and Drug Administration (FDA) granted Priority Review designation to belzutifan (MK-6482), an HIF-2α inhibitor, for the potential treatment of patients with von Hippel-Lindau disease–associated renal cell carcinoma who do not require immediate surgery.
The designation is based on the open-label phase II Study-004 trial data. The study enrolled 61 patients with von Hippel-Lindau disease who had at least one measurable solid tumor localized to the kidneys and who did not require immediate surgery. They received 120 mg orally once daily until disease progression or unacceptable toxicity. The primary endpoint was the objective response rate in von Hippel-Lindau disease–associated renal cell carcinoma. Secondary endpoints in renal cell carcinoma tumors included disease control rate, duration of response, time to response, progression-free survival, time to surgery, and safety. Approximately 22 patients had a confirmed overall response rate of 36.1% (95% confidence interval = 24.2%–49.4%) in patients with von Hippel-Lindau disease–associated renal cell carcinoma.
In addition to the ongoing phase II Study-004 clinical trial, belzutifan is being evaluated in phase III trials as a monotherapy and as part of a combination regimen in previously treated patients and part of a combination regimen a first-line treatment for advanced clear cell renal cell carcinoma. For more on this agent in combination with cabozantinib in metastatic clear cell kidney cancer, see here.