On May 14, 2025, the Food and Drug Administration (FDA) approved belzutifan (Welireg) for adult and pediatric patients 12 years of age and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL).[1] It is the first FDA-approved oral treatment for PPGL.[1] Efficacy was assessed in the open-label, multi-cohort study LITESPARK-015 (NCT04924075).[1] Cohort A1 included 72 patients with detectable disease confirmed by blinded independent central review (BICR) according to RECIST v1.1, a histologically confirmed diagnosis of PPGL, and locally advanced or metastatic disease not amenable to surgery or curative intervention.[1] The main measure of effectiveness was objective response rate (ORR) assessed by BICR according to RECIST v1.1.[1] Additional measures included duration of response (DOR) and the number of patients who achieved at least a 50% reduction in one antihypertensive drug level sustained for at least six months.[1]