FDA Approves Belzutifan for Advanced Renal Cell Carcinoma

The FDA has approved belzutifan (Welireg) for the treatment of patients with advanced renal cell carcinoma (RCC) following a PD-1 or PD-L1 inhibitor and a VEGF TKI.

Efficacy was evaluated in the randomized phase 3 LITESPARK-005 trial (NCT04195750), wherein belzutifan led to a statistically significant improvement in progression-free survival (PFS) vs everolimus (Afinitor) (HR, 0.75; 95% CI, 0.63-0.90; 1-sided P =.0008). Median PFS was 5.6 months (95% CI, 3.9-7.0) with belzutifan vs 5.6 months (95% CI, 4.8-5.8) with everolimus.

Overall survival (OS) data remain immature at the current analysis, with 59% of deaths reported. However, no trend towards a detriment was reported with belzutifan. A descriptive analysis of patient-reported symptom and functional outcomes also favored belzutifan compared with everolimus.

The open-label LITESPARK-005 trial enrolled 746 patients with unresectable locally advanced or metastatic clear cell RCC that had progressed after treatment with a PD-1 or PD-L1 inhibitor and a VEGF TKI. Patients were randomly assigned to receive 120 mg of belzutifan or 10 mg of everolimus once daily. Randomization was stratified by International Metastatic RCC Database Consortium risk category and number of prior VEGF TKIs.

The coprimary end points of the trial were PFS assessed by blinded independent central review and overall survival (OS).

The most common adverse effects that occurred in at least 25% of patients receiving belzutifan were decreased hemoglobin, fatigue, musculoskeletal pain, increased creatinine, decreased lymphocytes, increased alanine aminotransferase, decreased sodium, increased potassium, and increased aspartate aminotransferase.

The recommended dose of belzutifan is 120 mg administered orally once daily until disease progression or unacceptable toxicity.

Reference

FDA approves belzutifan for advanced renal cell carcinoma. FDA. December 14, 2023. Accessed December 14, 2023. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-advanced-renal-cell-carcinoma?utm_medium=email&utm_source=govdelivery