Merck's HIF-2α Inhibitor Welireg® (Belzutifan) Approved in China for VHL-Associated Tumors
MSD
Pharmaceutical R&D and Manufacturer
Shanghai, China,2024Year11Month21Day——MSD (the corporate name of Merck & Co., Inc., located in Rahway, New Jersey, U.S.) announced that its HIF-2α inhibitor Welireg® (belzutifan) has been approved by the China National Medical Products Administration (NMPA) for the treatment of adult patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET) who do not require immediate surgical intervention.
VHL disease, also known as von Hippel-Lindau syndrome or Lindau syndrome, is an autosomal dominant genetic disorder caused by mutations in the VHL tumor suppressor gene.[1], with an incidence rate of 1/91,000 to 1/36,000[1], [2], [3], [4]In China, the cumulative penetrance of patients reaches up to 97% at the age of 60.[1], [5]Patients with VHL disease exhibit a multi-organ tumor syndrome.[1]In China, the top three organs affected in VHL disease patients are the central nervous system (61.3%), pancreas (46.7%), and kidneys (42.7%), presenting as hemangioblastomas of the central nervous system, renal cell carcinoma or renal cysts, and pancreatic tumors or cysts, etc.[1], [6]。
"VHL disease is a hereditary and rare disease with a poor prognosis. The inactivation of VHL protein caused by gene mutations leads to the upregulation of downstream substrates such as hypoxia-inducible factor (HIF-α), which in turn promotes the expression of a series of carcinogenic factors, representing the main pathogenic mechanism of VHL disease."[1], [7]Surgery is one of the main treatment methods for such patients, but it is difficult to cure radically. Recurrence and even metastasis may still occur, and there is a lack of systematic treatment methods.[1]。”Professor Gong Kan, Department of Urology, Peking University First Hospital"We are very excited to see that Belzutifan has been approved for use in China, which will provide a new treatment option for patients with VHL disease, marking an important milestone in the development of VHL disease treatment."
"Viliri"®"As the first and only HIF-2α inhibitor approved in China, it will provide patients with a new mechanism of treatment options in this rare disease field, driving breakthroughs in the diagnosis and treatment of related diseases."Dr. Zhengqing Li, Senior Vice President of MSD Global and President of MSD China R&D CenterMSD said, "MSD is always committed to bringing high-quality drugs and vaccines to patients in China. This time, VELIRI®...®"Approval once again highlights our leading position in the field of oncology research and innovation, as well as our commitment and determination to meet the health needs of the Chinese public."
About MSD
At MSD (the corporate name of Merck & Co., Inc. in New Jersey, U.S.), we unite in pursuit of a shared mission: harnessing the power of leading-edge science to save and improve lives around the world. For over 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We are committed to being a top research-intensive biopharmaceutical company — today, we stand at the forefront of research, delivering innovative solutions to advance the prevention and treatment of diseases in humans and animals. We have built a diverse and inclusive global workforce, operating responsibly every day to ensure a safe, sustainable, and healthy future for all people and communities. For more information, visit www.msd.com and follow us on X (formerly Twitter), LinkedIn, and YouTube.
About MSD China
China is a crucial part of MSD's global growth strategy. MSD’s China headquarters is located in Shanghai, with a research and development center in Beijing and manufacturing facilities in Hangzhou, Ningbo, and Tianjin, integrating R&D, manufacturing, and commercial operations. We are fully committed to providing high-quality innovative medicines, vaccines, and services to the people of China, benefiting Chinese society. For more information, please visit MSD China's official website or follow MSD China's official social media accounts on WeChat.
MSD Forward-Looking Statements
MSD is the corporate name of Merck & Co., Inc., located in Rahway, New Jersey, USA (hereinafter referred to as "the Company"). This press release contains "forward-looking statements" made under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. The content herein is based on the current views and expectations of the Company’s management and is subject to significant risks and uncertainties. MSD does not guarantee that its products under development will receive the necessary regulatory approvals or achieve commercial success. If underlying assumptions prove inaccurate or risks or uncertainties arise, actual results may differ materially from those anticipated in the forward-looking statements.
Risks and uncertainties include, but are not limited to, general industry conditions and competition, general economic factors (including interest rate and currency fluctuations), the impact of pharmaceutical industry regulations and healthcare policies in the United States and other countries, the global trend toward controlling healthcare costs, technological advancements, new products and patents obtained by competitors, inherent challenges in new product development (including obtaining regulatory approval), MSD's ability to accurately predict future market conditions, difficulties or delays in production, instability in international economic and financial conditions and sovereign risks, reliance on the effectiveness of MSD’s patent and other innovative product protections, and the risk of patent litigation and/or regulatory actions against the company.
MSD has no obligation to publicly update any forward-looking statements due to new information, future events, or other reasons. Other factors may cause actual results to materially differ from forward-looking statements; see MSD's 2023 Annual Report on Form 10-K and other documents filed by the company with the U.S. Securities Exchange Commission (available at www.sec.gov).
[1]Beijing Medical Association Rare Disease Branch. Chinese Expert Consensus on the Diagnosis and Treatment of von Hippel-Lindau Disease [J]. Chinese Medical Journal, 2018, 98(28): 2220-2224
[2] E R Maher , L Iselius, J R Yates, et al. Von Hippel-Lindau disease: a genetic study[J]. J Med Genet. 1991 Jul;28(7):443-7. doi: 10.1136/jmg.28.7.443.
[3] I R Maddock, A Moran, E R Maher,et al. A genetic register for von Hippel-Lindau disease[J]. J Med Genet. 1996 Feb;33(2):120-7. doi: 10.1136/jmg.33.2.120.
[4] D.G. Evans,E. Howard, C. Giblin.et al. Birth Incidence and Prevalence of Tumor-Prone Syndromes: Estimates From a UK Family Genetic Register Servic[J]. Am J Med Genet A. 2010 Feb;152A(2):327-32.
doi: 10.1002/ajmg.a.33139.
[5] Jiang-Yi Wang , Shuang-He Peng , Teng Li,et al. Risk factors for survival in patients with von Hippel-Lindau disease[J]. J Med Genet. 2018 May;55(5):322-328. doi: 10.1136/jmedgenet-2017-04995. Epub 2018 Jan 12.
[6] Jiang-Yi Wang , Shuang-He Peng , Xiang-Hui Ning，et al. Shorter telomere length increases age-related tumor risks in von Hippel-Lindau disease patients[J].
[7] Lucy Gossage , Tim Eisen, Eamonn R Maher.VHL, the story of a tumour suppressor gene[J]. Nat Rev Cancer. 2015 Jan;15(1):55-64. doi: 10.1038/nrc3844.