FDA Approves Belzutifan for Advanced Renal Cell Carcinoma with prior therapy.

By: Dr. Anish Shah
Bronx-Lebanon Hospital; Bronx, NY


On December 14, 2023, the U.S. Food and Drug Administration granted approval to belzutifan (Welireg, Merck & Co., Inc.) for patients with advanced renal cell carcinoma (RCC) who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).


Study ID number: NCT04195750


Approval was based on LITESPARK-005, an open-label, randomized trial that included 746 patients with unresectable locally advanced or metastatic clear cell RCC that had progressed following both a PD-1 or PD-L1 checkpoint inhibitor and a VEGF-TKI. Patients were randomized to receive 120 mg belzutifan or 10 mg everolimus once daily. Belzutifan was given orally at a dose of 120 mg daily until disease progression or unacceptable toxicity.


Efficacy was established on progression-free survival (PFS) and overall survival (OS). Belzutifan showed a notable increase in progression-free survival (PFS) compared to everolimus, exhibiting a hazard ratio of 0.75 [(95% CI: 0.63, 0.90); 1-sided p-value=0.0008]. The Kaplan-Meier analysis revealed non- proportional hazards with comparable median PFS projections of 5.6 months for both belzutifan and
everolimus. Although only 59% of the overall survival (OS) data had been analyzed at the time, there was no discernible pattern indicating any adverse impact.


The most common adverse reactions in patients receiving belzutifan were anemia, fatigue, musculoskeletal pain, kidney injury, decreased lymphocytes, increased alanine aminotransferase, decreased sodium, increased potassium, and increased aspartate aminotransferase.


The FDA approved belzutifan for advanced RCC patients post PD-1 or PD-L1 and VEGF-TKI inhibitor treatment. The recommended dose is 120 mg orally daily until disease progression or unacceptable toxicity.


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