China’s National Medical Products Administration (NMPA) has approved MSD’s hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, oral WELIREG (belzutifan), to treat adults with certain types of von Hippel-Lindau (VHL) disease-associated tumours.
These include renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), which do not require immediate surgery.
The approval of WELIREG in China is based on the Phase II LITESPARK-004 trial’s objective response rate (ORR) and median duration of response (DOR) outcomes, and is the 17th global approval for this indication. WELIREG was approved in the US in August 2021 for similar indications.
The open-label trial enrolled 61 subjects with VHL-associated RCC and demonstrated an ORR of 49% with all responses being partial responses. Median DOR was not attained, with ongoing responses lasting from 2.8 to more than 22 months.
Patients with other VHL-associated tumours, including CNS hemangioblastomas and pNET, also showed significant response rates in the LITESPARK-004 trial.
CNS hemangioblastomas patients had an ORR of 63%, with a complete response rate of 4% and a partial response rate of 58%.
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Patients with VHL-associated pNET exhibited an ORR of 83%, with a complete response rate of 17% and a partial response rate of 67%.
MSD research laboratories oncology, global clinical development head and senior vice-president Dr Marjorie Green stated: “This approval of WELIREG brings the first and only systemic therapy to adult patients in China with certain VHL disease-associated tumours who, to date, have not had access to a non-surgical treatment option to help manage manifestations of VHL disease.”
WELIREG has been approved in the US for the treatment of adult patients with advanced RCC after 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), based on the Phase III LITESPARK-005 trial outcomes.
MSD is further assessing the therapy in advanced RCC and other tumour types via a comprehensive clinical development programme, including in Phase II and III trials as a monotherapy and in conjunction with other medicines.
The company recently secured a worldwide licence for LaNova Medicines’ LM-299.
