FDA Grants Priority Review to Merck’s sNDA for Welireg (belzutifan) in Advanced Renal Cell Carcinoma

▎WuXi

Edited by Kant Content Team

MSD announced today,The US FDA has accepted the supplemental New Drug Application (sNDA) for its “first-in-class” hypoxia-inducible factor-2α (HIF-2α) inhibitor Welireg (belzutifan) and granted it priority review status., for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have experienced disease progression after receiving immune checkpoint and anti-angiogenic therapies. The FDA is set to complete its review by January 17, 2024.

Renal cell carcinoma is the most common type of kidney cancer, accounting for about 9 out of 10 kidney cancer diagnoses. The incidence in men is approximately twice that in women. Most cases are incidentally discovered during imaging tests for other abdominal conditions. About 15% of patients with kidney cancer are diagnosed at an advanced stage.

Welireg is the first HIF-2α inhibitor to receive FDA accelerated approval., which has been approved in the United States, the United Kingdom, Canada, and several other countries and regions based on the objective response rate (ORR) and duration of response (DOR) data from the Phase 2 LITESPARK-004 trial, for the treatment of adult patients with von Hippel-Lindau (VHL) disease who have related renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors and do not require immediate surgery.

The sNDA submission is primarily based on data from the LITESPARK-005 trial. A pre-specified interim analysis conducted by an independent data monitoring committee showed that, in patients with advanced RCC whose disease progressed after receiving PD-1/PD-L1 checkpoint inhibitors and vascular endothelial growth factor tyrosine kinase inhibitors (VEGF-TKI), compared to everolimus,Patients in the Welireg group experienced a statistically significant and clinically meaningful improvement in progression-free survival (PFS). The key secondary endpoint, objective response rate (ORR), also showed a statistically significant improvement.

References:

[1] FDA Accepts for Priority Review Merck’s Supplemental NewDrug Application for WELIREG® (belzutifan) in Certain Previously TreatedPatients With Advanced Renal Cell Carcinoma (RCC). Retrieved September 19, 2023from https://www.merck.com/news/fda-accepts-for-priority-review-mercks-supplemental-new-drug-application-for-welireg-belzutifan-in-certain-previously-treated-patients-with-advanced-renal-cell-carcinoma-rcc/