The European medicines agency review of belzutifan (Welireg) for the treatment of adult patients with von Hippel–Lindau disease-associated tumors

In February 2025, the European Commission granted approval for belzutifan (Welireg) as a treatment for certain von Hippel-Lindau (VHL) disease-associated tumors. This is the first medicinal product in Europe for pharmacological management of VHL, a rare, hereditary condition characterized by the development of multiple tumors across various organs. Belzutifan is an oral inhibitor of hypoxia-inducible factor-2α (HIF-2α), a protein involved in the pathogenesis of VHL-associated tumors. This approval, which is based on the results of the pivotal phase 2 clinical trial MK-6482-004 (LITESPARK-004) offers a therapeutic approach to managing certain VHL disease-associated tumors. This article describes the clinical data supporting belzutifan approval in the EU, focusing on the VHL disease indication and its mechanism of action.