Belzutifan for von Hippel-Lindau Disease-Associated Neoplasms

This technical report presents updated results from the LITESPARK-004 study, a single-arm, phase 2 trial evaluating the efficacy of belzutifan, a hypoxia-inducible factor-2α inhibitor, for treating von Hippel-Lindau disease-associated renal cell carcinoma and other neoplasms. The study enrolled 61 participants across 11 centers in Denmark, France, the UK, and the USA, with a median follow-up of 49.9 months. The primary endpoint assessed the objective response rate in renal cell carcinoma, revealing that 67% of participants achieved an objective response, with 11% attaining a complete response. The report details the safety profile, noting that 18% of participants experienced grade 3 treatment-related adverse events, but no grade 4 or 5 events were reported. The findings support the continued use of belzutifan as an effective systemic treatment option, potentially reducing the need for surgical interventions in this patient population.