EC Grants Conditional Approval to MSD's Welireg for VHL Disease and RCC

The European Commission (EC) has granted conditional approval for MSD's Welireg, an oral inhibitor known as hypoxia-inducible factor-2 alpha (HIF-2α), to be used as a monotherapy for adults with von Hippel-Lindau (VHL) disease and advanced clear cell renal cell carcinoma (RCC). This decision marks a significant milestone, as it introduces the first systemic treatment option for VHL disease-associated tumors within the European Union (EU).

Welireg is specifically approved for adult patients with VHL disease who necessitate intervention for related renal cell carcinoma (RCC), central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors. Additionally, it caters to individuals for whom localized treatment methods are not feasible. Furthermore, the approval extends to adults with advanced clear cell RCC that has progressed following at least two prior lines of therapy, including those involving a programmed death-ligand 1 inhibitor or a programmed death receptor-1, alongside at least two treatments targeting vascular endothelial growth factor.

The EC's conditional approval is derived from results obtained in the LITESPARK-004 and LITESPARK-005 open-label clinical trials. This authorization remains valid for one year and necessitates annual renewal contingent upon the submission of supplementary clinical data from ongoing trials, particularly the Phase II LITESPARK-004 trial, which focuses on tumors associated with VHL disease.

Welireg is now recognized in over 30 countries for certain adult patients who have previously undergone treatment for advanced renal cell carcinoma and in more than 40 nations for individuals with specific tumors linked to VHL disease. With this approval, Welireg can now be marketed throughout all 27 EU member states, including Norway, Iceland, and Liechtenstein. The precise timing for the therapy's commercial availability within individual EU countries will depend on national reimbursement processes and other related factors.

This landmark decision reinforces Welireg's position as a groundbreaking option, providing hope and a new line of treatment for patients dealing with the challenges of VHL disease-associated tumors. The approval is based on the impressive outcomes related to the duration of response and objective response rate documented in the LITESPARK-004 trial, offering an innovative therapeutic avenue for patients who previously had limited options.