September 9, 2020, Beijing, China; Cambridge, Massachusetts--CANbridge Pharmaceuticals Inc., a company developing and commercializing innovative drugs to treat rare diseases and targeted cancers, announced that it received marketing approval from China’s National Medical Products Administration (NMPA) for Hunterase® (Idursulfase beta Injection), an enzyme replacement therapy (ERT) for the long term treatment of patients with mucopolysaccharidosis II (MPS II or Hunter syndrome). This is the first approved treatment in the CANbridge rare disease portfolio and the first ERT treatment in China for Hunter syndrome, where it fills an urgent treatment gap. Developed by GC Pharma (KRX: 006280), Hunterase is designated an orphan drug in the United States and Korea and is marketed in 11 countries. CANbridge obtained the exclusive licensing rights for Hunterase from South Korea’s GC Pharma in January, 2019 to commercialize the drug in greater China, where it was granted priority review by NMPA in September, 2019.
“The approval of our first rare disease treatment is a major step toward fulfilling our mission to develop and commercialize treatments for rare diseases, which are desperately needed in China and the rest of the world,” said James Xue, PhD, CANbridge Founder, Chairman and CEO. “The launch of Hunterase is the result of the joint efforts of our industry partners, including GC Pharma, which conducted the overseas clinical trials and medical experts, as well as the MPS II patients and families who took part in the clinical trials and played a crucial role in bringing Hunterase to China. We are encouraged by their unremitting efforts, which inspire us to continue our work to develop rare disease treatments and build the rare disease ecosystem in China.”
“There is an urgent need for effective therapeutic drugs for this genetic disease that seriously endangers the life of patients,” said Xuefan Gu, PhD, a member of the Committee of Experts on the Diagnosis and Treatment of Rare Diseases and the Guarantee of Drug Supplies of the National Health Commission, Director of the Shanghai Children’s Rare Diseases Center and Professor of Xinhua Hospital, affiliated with the Shanghai Jiaotong University School of Medicine.
About Hunter syndrome (MPS II)
Hunter syndrome (MPS II) is an inherited lysosomal storage disease that is X-linked and occurs primarily in boys. It causes an enzyme deficiency that interferes with the body’s ability to break down certain complex sugar polymers called glycosaminoglycans, resulting in serious skeletal, tissue, neurological and multi-organ complications and, ultimately, a shortened lifespan. The median age of onset is under 5 years and the median age of death, typically from heart or respiratory failure, is 15, according to Xuefan Gu, PhD, Director of the Shanghai Children’s Rare Diseases Center.
Hunter syndrome occurs in approximately 0.13-2.16/100,000 live births in Europe, but has a significantly higher incidence rate in East Asian countries and regions, occurring in approximately 1.07/ 100,000 live births in Taiwan1. There is no cure currently, but enzyme replacement therapy (ERT) is unanimously recommended as the standard treatment for MPS II2,3,. If started early, ERT can slow down, or even prevent, disease progression, according to Professor Xiaoping Luo, Vice President, Chinese Pediatric Society, CMA. Mucopolysaccharidosis is listed as No. 73 in China’s national “List of Rare Diseases”4
According to the 2020 report, “Analysis of the Survival of Chinese Rare Disease Patients from the Perspective of Mucopolysaccharidos,” 41% of patients with mucopolysaccharidosis in China did not receive any treatment after diagnosis, while 47% of them were only treated symptomatically, due to lack of ERT in China. 8.95% of patients travelled overseas to receive effective ERT treatment. Specifically, 29 MPS II patients and their families traveled to South Korea, through the Beijing Zhengyu Mucopolysaccharide Rare Disease Care Center, in the past few years to participate in Hunterase clinical trials 5.
Hunterase®, an injection of iduronidase-2-sulfatase(IDS): recombinant human iduronidase-2-sulfatase, is used for patients with Hunter syndrome above 38 months of age and with varying degrees of disease severity.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. is a biopharmaceutical company accelerating development and commercialization of specialty healthcare products for orphan diseases and targeted cancers to address unmet medical needs.
CANbridge has a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases. In greater China, where it is a recognized leader in orphan diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome, developed by GC Pharma and marketed in more than 11 countries worldwide. CANbridge also has a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases. The CANbridge oncology portfolio includes exclusive rights to develop and commercialize Puma Biotechnology’s NERLYNX® (neratinib), which was recently approved in greater China, as well as rights to other novel candidates.
For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.
1.Molecular Genetics and Metabolism, 2017
2.Scarpa M, et al. Orphanet J Rare Dis. 2011 Nov 7;6:72
3.Muenzer J, et al. Eur J Pediatr. 2012;171:181-188.
4.A list of rare diseases, jointly issued by China‘s National Health Commission and five other departments in May 2018, which includes 121 rare diseases.
5.QQ.COM, 15 May 2020, xw.qq.com/cmsid/20200515A06GUW00
CANbridge Pharmaceuticals Inc.