Originally published by our sister publication Clinical Oncology News
By Clinical Oncology News Staff
The FDA approved belzutifan (Welireg, Merck) for adult and pediatric patients aged 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or 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 (BICR) per RECIST version 1.1, documented histopathologic PPGL diagnosis, and locally advanced or metastatic disease not amenable to surgery or curative treatment. Patients with concomitant hypertension with adequately controlled blood pressure were required to have no change in antihypertensive medications for at least two weeks prior to study initiation. Patients with carcinomatous meningitis were excluded.
The major efficacy outcome measure was objective response rate (ORR) by BICR using RECIST version 1.1. Additional efficacy outcome measures were duration of response (DOR) and the number of patients who had a reduction in at least one antihypertensive medication by at least 50% maintained for at least six months. The ORR was 26% (95% CI, 17%-38%). Median DOR was 20.4 months (95% CI, 8.3 months to not reached). Of the 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 six months.
The most common adverse reactions (≥25%), including laboratory abnormalities, were 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.
The full prescribing information for belzutifan is posted on Drugs@FDA.
Based on a press release from the FDA.
