Merck's Welireg approved for cancers associated with von Hippel-Lindau disease in China

Published: 25-Nov-2024

The once-daily drug is the first HIF-2α to be approved in China, exhibiting a 49% objective response rate (ORR)

Merck has received approval for Welireg from the National Medical Products Administration (NMPA) of China.

The therapeutic, otherwise called belzutifan, is suitable for adult patients with von Hippel-Lindau (VHL) disease who have associated cancers, including renal cell carcinoma, CNS haemangioblastomas or pancreatic neuroendocrine tumours.

It is an oral HIF-2α inhibitor, and is the first to gain approval in China.

Welireg's approval is based on results from the Phase II LITESPARK-004 trial, which found that the ORR and median duration of response (DOR) are significant in this patient population. 

During the LITESPARK-004 clinical study, Welireg demonstrated an ORR of 49%, in patients with renal cell carcinomas, while 56% of patients maintained a response to the therapeutic for at least 12 months — though ongoing responses ranged from 2.8 to 22.3 months.

This effect was also seen in patients with CNS hemangioblastomas, where the ORR was 63%.

Welireg has also gained approved in the US, and can be used to treat adult patients with advaned renal cell carcinoma following administration of both PD-L1 inhibitors and VEGF tyrosine kinase inhibitors.

Merck is currently evaluating the efficacy of Welireg in other tumour types as a monotherapy and in combination with other agents. 

“This approval of WELIREG brings the first and only systemic therapy to adult patients in China with certain VHL disease-associated tumors who, to date, have not had access to a non-surgical treatment option to help manage manifestations of VHL disease,” said Dr Marjorie Green, Senior VP and Head of Oncology, Global Clinical Development, Merck Research Laboratories.

“We are committed to bringing innovative treatment options to patients in need around the world and are proud to offer eligible adult patients in China a first-in-class HIF-2α inhibitor as a possible treatment option.”

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