NMPA approves MSD's WELIREG for VHL-associated tumours

China’s National Medical Products Administration (NMPA) has approved the use of MSD's oral WELIREG (belzutifan) for the treatment of adults with specific types of von Hippel-Lindau (VHL) disease-associated tumors. These include renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, and pancreatic neuroendocrine tumors (pNET), particularly in cases where immediate surgery is not required.

This approval in China is based on the findings from the Phase II LITESPARK-004 trial, which evaluated the objective response rate (ORR) and median duration of response (DOR). As a result, China becomes the 17th country to approve WELIREG for this indication. Previously, in August 2021, the drug received approval in the United States for similar uses.

The LITESPARK-004 trial, an open-label study, involved 61 participants with VHL-associated RCC. The trial revealed an ORR of 49%, with all responses being partial responses. The median DOR had not been reached, with responses lasting from 2.8 months to over 22 months.

Significant responses were also observed in patients with other VHL-associated tumors, such as CNS hemangioblastomas and pNET, during the LITESPARK-004 trial. The ORR for patients with CNS hemangioblastomas was 63%, including a complete response rate of 4% and a partial response rate of 58%. For patients with VHL-associated pNET, the ORR was 83%, comprising a complete response rate of 17% and a partial response rate of 67%.

Dr. Marjorie Green, head of MSD research laboratories oncology, global clinical development, and senior vice-president, remarked on the significance of this approval. She noted that WELIREG is the first systemic therapy available to adults in China with specific VHL disease-associated tumors, providing a non-surgical treatment option for managing the disease.

WELIREG is also approved in the US for treating adult patients with advanced RCC, particularly after prior treatment with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor-tyrosine kinase inhibitor (VEGF-TKI). This approval was based on results from the Phase III LITESPARK-005 trial.

MSD is continuing to explore the potential of WELIREG in treating advanced RCC and other tumor types through an extensive clinical development program. This includes conducting Phase II and III trials, both as a standalone therapy and in combination with other treatments.