FDA Grants Priority Review to Belzutifan in Previously Treated Advanced RCC

By Zachary Bessette - Last Updated: September 20, 2023

The US Food and Drug Administration (FDA) has accepted for priority review the supplemental new drug application for Merck’s WELIREG (belzutifan) in patients who were previously treated for advanced renal cell carcinoma (RCC).

The acceptance of this priority review is based on results from the phase 3 LITESPARK-005 trial, which were read out in August and showed a statistically significant and clinically meaningful improvement in progression-free survival compared with everolimus in patients with advanced RCC who experienced disease progression after PD-1/PD-L1 and VEGF-TKI therapies.

A statistically significant improvement in objective response rate, the trial’s key secondary end point, was also reported.

“Patients with advanced RCC whose cancer progresses following immune checkpoint and anti-angiogenic therapies face a poorer prognosis, and for those patients, there is a crucial unmet need for new options with an alternative mechanism of action,” said Marjorie Green, MD, senior vice president and head of late-stage oncology, global clinical development, at Merck, in a press release. “The FDA’s priority review designation of this application reinforces the urgency to provide new options to previously treated patients with advanced RCC.”

Belzutifan was the first HIF-2α inhibitor approved in the United States. It is currently indicated for patients with von Hippel-Lindau disease who require therapy for associated RCC, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors.

The HIF-2α inhibitor is being further assessed in LITESPARK-011 and LITESPARK-012 as a second-line therapy for treatment-naïve advanced disease, and in LITESPARK-022 as an adjuvant therapy option.