FDA Priority Review for New Merck Welireg Indication

FDA has accepted for priority review a Merck sNDA for a new indication for its Welireg (belzutifan) to treat adults and pediatric patients over age 12 with advanced pheochromocytoma and paraganglioma. A company statement says the sNDA was based on objective response rate and duration of response data from the Phase 2 LITESPARK-015 trial. It says FDA set 5/26 as the PDUFA action date for the sNDA.

Welireg is described as the only HIF-2a inhibitor therapy approved in the U.S. to treat adults with von Hippel-Landau disease who require therapy for associated renal cell carcinoma (RCC), central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors not requiring immediate surgery, based on results from the Phase 2 LITESPARK-004 trial. It also is approved to treat adults with advanced RCC following a PD-1/PD-L1 inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor, based on results from the Phase 3 LITESPARK-005 trial.