FDA Approves Welireg for Pheochromocytoma and Paraganglioma

The FDA approved belzutifan (Welireg, Merck) for adult and pediatric patients aged 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL). This represents the first FDA approval of an oral therapy for PPGL.

Efficacy was evaluated in LITESPARK-015 (ClinicalTrials.gov Identifier: NCT04924075), an open-label, multi-cohort trial. Cohort A1 was conducted in 72 patients with measurable disease verified by blinded independent central review