Officials with the FDA recently approved belzutifan (Welireg; Merck) for people aged ≥12 years with locally advanced unresectable or metastatic pheochromocytoma or paraganglioma (PPGL), making it the first FDA approval of an oral therapy for PPGL.1
Researchers evaluated the therapy in LITESPARK-015 (NCT04924075), an open-label, multicohort trial. Cohort A1 was conducted in 72 patients with measurable disease verified by a blinded independent central review per RECIST v1.1, documented histopathological PPGL diagnosis, and locally advanced or metastatic disease not amenable to surgery or curative treatment.
The researchers noted the median objective response rate was 26% (95% confidence interval [CI], 17-38). Median duration of response was 20.4 months (95% CI, 8.3-not reached). Of 60 patients on baseline antihypertensive medications, 19 (32%; 95% CI, 20-45) had a reduction in at least one antihypertensive medication by at least 50% for at least 6 months.
The most common adverse reactions included anemia, fatigue, musculoskeletal pain, decreased lymphocytes, increased alanine aminotransferase, increased aspartate aminotransferase, increased calcium, dyspnea, increased potassium, decreased leukocytes, headache, increased alkaline phosphatase, dizziness, and nausea.
Reference
- FDA. FDA approves belzutifan for pheochromocytoma or paraganglioma [press release]. May 14, 2025. Accessed July 1, 2025. www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-pheochromocytoma-or-paraganglioma