VHL Europa, representing the voice of von Hippel-Lindau (VHL) patients across Europe, is thrilled by the news that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the conditional approval of WELIREG® (belzutifan). If approved, belzutifan will become the first systemic treatment available for adult patients with VHL-associated tumors in the European Union. Here is the official message: https://www.ema.europa.eu/en/medicines/human/EPAR/welireg
This landmark decision brings hope to the European VHL community, as it marks a significant step forward in the management of VHL disease. For years, patients and their families have faced the challenges of this multi-system genetic disorder, which often necessitates repeated surgical interventions. The availability of an oral treatment specifically targeting hypoxia-inducible factor-2 alpha (HIF-2α) offers a much-needed therapeutic alternative, potentially improving quality of life and disease outcomes.
The positive opinion is based on robust data from the Phase 2 LITESPARK-004 trial and the Phase 3 LITESPARK-005 trial, underscoring the efficacy and safety of belzutifan for treating localised renal cell carcinoma (RCC), central nervous system (CNS) haemangioblastomas, and pancreatic neuroendocrine tumors (pNET) associated with VHL.
VHL Europa applauds the efforts of researchers, clinicians, and industry partners who have contributed to this breakthrough and eagerly anticipates the European Commission’s final decision.
For more information, please contact VHL Europa at info@vhl-europa.org.
About VHL Europa:
VHL Europa represents European patients and families affected by von Hippel-Lindau disease, advocating for access to comprehensive care and innovative therapies. Through collaboration with healthcare providers and industry leaders, VHL Europa is committed to improving outcomes for those living with this rare genetic condition.