Merck’s Welireg sNDA accepted for priority review in PPGL

The US Food and Drug Administration (FDA) has accepted for priority review a supplemental new drug application (sNDA) for Welireg (belzutifan), Merck & Co’s (NYSE: MRK) oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, for the treatment of adult and pediatric patients (12 years and older) with advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL).

The sNDA is based on objective response rate (ORR) and duration of response (DOR) data from the Phase II LITESPARK-015 trial, which will be presented at an upcoming medical meeting. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of May 26, 2025.

“Pheochromocytoma and paraganglioma are rare tumors that form in and around the adrenal glands, and currently, there are no approved therapies available in the US for patients with this rare disease,” said Dr Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. “Today’s U.S. filing acceptance demonstrates our commitment to advancing novel therapies, such as Welireg, to help treat patients with certain rare oncologic diseases. We look forward to working with the FDA to potentially provide this critical option to these patients who urgently need new innovative therapies,” she noted.