The U.S. Food and Drug Administration (FDA) has granted priority review to Merck’s application for WELIREG® (belzutifan) as a treatment for adult and pediatric patients (12 years and older) living with advanced pheochromocytoma and paraganglioma (Pheo/Para). The FDA aims to decide about belzutifan by May 26, 2025.
Belzutifan is a novel oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor approved in the U.S. to treat certain tumors associated with von Hippel-Lindau (VHL) disease. The potential new indication for Pheo/Para could expand belzutifan’s use, providing hope to many facing these challenging diagnoses. If approved, the drug would be the only available therapy in the U.S. for eligible patients with advanced Pheo/Para.
The FDA’s priority review decision comes after encouraging results from the Phase 2 LITESPARK-015 trial, which demonstrated promising objective response rates and duration of response among participants.
LITESPARK-015 is an open-label, single-arm, multi-cohort Phase 2 trial evaluating the efficacy and safety of WELIREG monotherapy in patients with advanced, pancreatic neuroendocrine tumors, VHL disease-associated tumors, advanced gastrointestinal stromal tumors, or advanced solid tumors with HIF-2α-related genetic alterations.
“Pheochromocytoma and paraganglioma are rare tumors that significantly impact patients’ lives, often requiring complex management strategies,” said Dr. Marjorie Green, senior vice president and head of oncology global clinical development at Merck Research Laboratories. “The acceptance of this application by the FDA brings us closer to potentially providing a critical new treatment option for these patients.”