US pharmaceutical giant Merck, Sharp & Dohme (MSD, NYSE: MRK) announced that it has received conditional approval from the European Commission (EC) for its oral drug Welireg (belzutifan) for the treatment of von Hippel-Lindau (VHL) disease and advanced clear cell renal cell carcinoma (RCC).
Drug Profile
Welireg is a hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor. The EC approved it as a monotherapy for adult patients with VHL disease requiring treatment for localized RCC, central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), when localized procedures are unsuitable. It is also approved for adult patients with advanced clear cell RCC that progressed after two or more lines of therapy, including a PD-1 or PD-L1 inhibitor and at least two VEGF targeted therapies.
Clinical Trial Results
- VHL Disease: The approval is based on the LITESPARK-004 study, where Welireg demonstrated an objective response rate (ORR) of 49% in VHL-associated RCC, 63% in CNS hemangioblastomas, and 83% in pNET patients.
- Advanced RCC: The LITESPARK-005 study showed that Welireg reduced the risk of disease progression or death by 25%, with an ORR of 22% compared to 4% for everolimus.
Future Outlook
Welireg is now the first and only systemic therapy for VHL disease-associated tumors in the European Union. The conditional approval highlights the drug’s potential to address unmet medical needs in rare genetic disorders and advanced cancers.-Fineline Info & Tech
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