WELIREG: First Systemic Therapy Approved for VHL Disease in China

WELIREG Approved in China for VHL Disease

November 22, 2024 – 6:45 AM ET

Merck announced that China’s National Medical Products Administration (NMPA) has approved WELIREG® (belzutifan) for treating adult patients with von Hippel-Lindau (VHL) disease. This marks the first systemic therapy approved for certain VHL-associated tumors in China.

Key Highlights:

• WELIREG is the only approved HIF-2α inhibitor in China.
• The approval is based on results from the Phase 2 LITESPARK-004 trial.
• This is the 17th global approval of WELIREG for these patients.

Dr. Marjorie Green from Merck stated that this approval provides patients with a much-needed non-surgical treatment option.

Clinical Trial Insights:

• The LITESPARK-004 trial involved 61 patients with VHL-associated tumors.
• For renal cell carcinoma (RCC), WELIREG showed an objective response rate (ORR) of 49%.
• In patients with CNS hemangioblastomas, the ORR was 63%.
• In pancreatic neuroendocrine tumors (pNET), the ORR was 83%.

WELIREG’s Indications:

• Treatment for adult patients with VHL disease and associated RCC, CNS hemangioblastomas, or pNET not requiring immediate surgery.
• Approved in the U.S. for advanced RCC after prior therapies.

Safety Information:

• WELIREG may cause severe anemia, hypoxia, and fetal harm if taken during pregnancy. Regular monitoring is necessary.
• Patients should be advised about the risks and the need for effective contraception.

About VHL Disease:
VHL disease is a rare genetic condition affecting around 200,000 people worldwide, leading to various tumors, primarily RCC.

For further details, visit Merck’s oncology research page or their main website.