In a breakthrough for those with advanced pheochromocytoma and paraganglioma (Pheo/Para), the U.S. Food and Drug Administration (FDA) has approved WELIREG® (belzutifan) as a treatment for adult and pediatric patients (12 years and older) with metastatic or inoperable tumors. The drug is the only oral therapy available for these patients.
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 new indication for Pheo/Para will expand belzutifan’s use, providing hope to many facing these challenging diagnoses.
The FDA’s approval comes after encouraging results from the Phase 2 LITESPARK-015 trial, which demonstrated promising objective response rates and duration of response among participants.
“This approval, which is based on objective response rate data from the LITESPARK-015 trial, introduces belzutifan as the only approved and available non-surgical option for locally advanced, unresectable, or metastatic PPGL and could represent a change to the treatment paradigm for eligible patients,” says Dr. Camilo Jimenez, professor, department of endocrine neoplasia and hormonal disorders, The University of Texas MD Anderson Cancer Center. More on the trial here.