Dr Rini on the FDA Approval of Belzutifan in Relapsed/Refractory RCC

Brian I. Rini, MD, FASCO, Ingram Professor of Medicine, Department of Medicine, Division of Hematology Oncology, Vanderbilt University, chief, Clinical Trials, Vanderbilt-Ingram Cancer Center, make sure the institution name is changed in the lower third too discusses the importance of the FDA approval of belzutifan (Welireg) in patients with relapsed/refractory renal cell carcinoma (RCC). 

On December 14th, 2023, the FDA approved belzutifan for the treatment of patients with RCC who have progressed on a previous PD-1 or PD-L1 inhibitor and a VEGF TKI. This regulatory decision was supported by data from the phase 3 LITESPARK-005 trial (NCT04195750), in which patients experienced a statistically significant improvement in progression-free survival (PFS) with belzutifan compared with everolimus (Afinitor; HR, 0.75; 95% CI, 0.63-0.90; 1-sided P = .0008). The 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.

With the approval of belzutifan, another effective treatment option is now available in the refractory RCC setting, Rini begins. It's always beneficial to have more active drugs; this approval is a positive development overall, he says. Each patient may respond differently to various treatments and tolerate them differently, making certain treatments more suitable for specific patients, Rini shares. Therefore, much work remains to be done to determine which patients benefit most from which therapies and how to apply these treatments most effectively, according to Rini. Future trials and continued clinical use of these agents may answer some of these questions, he explains.

Nonetheless, the addition of a well-tolerated, active drug is always welcome, particularly in the refractory kidney cancer setting, Rini continues. Looking ahead, apart from identifying disease subsets that are particularly susceptible to belzutifan treatment, ongoing trials are exploring combination regimens with this agent in both the second- and first-line settings, he adds. The tolerability of belzutifan makes it a promising candidate for combination regimens, although data on this are currently lacking, Rini elucidates.

Hopefully, the next couple years will provide more information about the efficacy of belzutifan in earlier lines of treatment and in combination regimens, potentially leading to its integration into earlier stages of the disease and various combination settings, Rini concludes.