First Single-Centre Experience with the Novel HIF-α Inhibitor Belzutifan in Switzerland

Belzutifan is a new HIF-α inhibitor mainly used in two different indications: von Hippel-Lindau syndrome-associated renal cell carcinoma, haemangioblastomas and pancreatic neuroendocrine tumours, as well as sporadic advanced pre-treated renal cell carcinoma. Although efficacy has been demonstrated in phase II and III studies, belzutifan is still not approved in many countries. In addition, von Hippel-Lindau syndrome is a rare disease. Therefore, there is virtually no real-world experience data of belzutifan efficacy available. We aim to determine the real-world efficacy and tolerability of belzutifan in patients with von Hippel-Lindau syndrome-associated tumours and in patients with sporadic advanced tyrosine kinase- and immune checkpoint inhibitors pre-treated for renal cell carcinoma. A retrospective analysis of five patients treated with belzutifan between 2023 and 2024 at a Swiss cancer centre was conducted. In this case series, all patients consistently benefitted from belzutifan with response to treatment. This case series provides real-world evidence that belzutifan is an effective and well-tolerated treatment option for patients with von Hippel-Lindau syndrome-associated renal cell carcinoma, haemangioblastomas and sporadic advanced pre-treated renal cell carcinoma.