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    Home > Active Ingredient News > Drugs Articles > China's first mucosal polysaccharide storage disease TYPE II. Enzyme replacement therapy Beihai Kangcheng rare disease new drug has been approved for market.

    China's first mucosal polysaccharide storage disease TYPE II. Enzyme replacement therapy Beihai Kangcheng rare disease new drug has been approved for market.

    • Last Update: 2020-09-26
    • Source: Internet
    • Author: User
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    On September 9, 2020, CANbridge Pharmaceuticals (CANbridge Pharmaceuticals) announced that its long-term enzyme replacement drug Hunterase ® (Adolfate Enzyme Beta Injection) for the treatment of mucos®al polysaccharide storing disorder TYPE II., also known as Hunter syndrome, has been approved by China's National Drug Administration.
    Hai weith® as the first rare disease drug successfully commercialized in China by Beihai Kangcheng, is the first and only global next-generation enzyme replacement therapy (Enzyme replace therapy, ERT), has FDA orphan drug qualification, and has been approved by the National Drug Administration for the diagnosis of mucosal polysaccharide storage syndrome II.
    to fill the unseeded demand for drug treatment in China, and ushered in a new era of treatment of MPS II.
    has been used ® 11 countries around the world and has been proven to be effective Chinese safety in a wide range of populations and groups.
    As the world's next generation of enzyme replacement therapy, Hyacinth ® significantly reduces uGAGs levels in MPS II patients, significantly improving the 6-minute walking test distance (6-MWT) and benefiting patients under 6 years of age.
    production process of ® haitsi is innovative, its enzyme activity is high, and the serum-free culture base avoids the risk of external contamination.
    the drug can be used for treatment, safe and stable, good infusion tolerance.
    to fill ® clinical gap in the treatment of MPSII. Enzyme replacement in China, is a rare progressive disability, fatal X chain recessive genetic disease.
    because of the absence or deficiency of Adolfalic acid-2-sulphatease (IDS), glycamine polysaccharides (GAG) stored in the patient's body can affect multiple organ systems.
    currently, mucosal polysaccharose storage disorder (MPS) has been included in the national "first list of rare diseases", ranked No. 73.
    "MPSII. The middle age of patients is mostly before the age of 5 years, the main clinical manifestations are stunting, rough face, bone deformities, liver swelling and so on.
    mPSII. Patients died of heart or respiratory diseases when they were under 15 years of age.
    is urgently needed for this seriously life-threatening genetic disease.
    Professor Gu Xuefan, Member of the Expert Committee on Rare Diseases diagnosis and treatment and protection of the National Health And Wellness Commission, Professor of Xinhua Hospital affiliated with Shanghai Xuanda Medical College, Director of the Shanghai Children's Rare Diseases Diagnosis and Treatment Center", according to the report "Analysing the Survival Status of Patients with Rare Diseases in China from Slim Polysaccharies Storage Disorders" released in 2020 Due to the lack of effective enzyme replacement therapy drugs in China, 41% of patients with mucosal polysaccharide storing disorder did not receive any treatment after diagnosis, 47% of patients can only treat the disease, and even 8.95% of patients in order to receive effective drug treatment and go overseas.
    enzyme replacement therapy (ERT) is unanimously recommended as the standard treatment for MPSII.
    "MPSII. Caused by organ system damage is almost irreversible, if the early stage of the disease can be given timely treatment, so that patients receive long-term standardized enzyme replacement therapy, can prevent and delay the progression of the disease, but also can avoid the emergence of serious complications, improve the multi-organ system burden, improve growth and development, give patients hope for life.
    approval of the ® market will change the plight of mpSII. Patients in China have no cure.
    In the past few years, 29 Chinese MPSII. patients, coordinated by the Center and GC Pharma, have participated in overseas clinical trials of Hyo-si ®.
    , we are finally looking forward to the introduction of this drug to China by North Sea Kangcheng, and obtained the country's market approval.
    we eagerly hope that all MPSII patients will be able to use the drug at home as soon as possible and continue to write the miracle of life.
    Zheng Tuo, Director of Beijing Zhengyu Sticky Polysaccharide Rare Disease Care Center, "Beihai Kangcheng is committed to the development and commercialization of china's rare disease patients in urgent need of special-effects rare drugs."
    the successful listing of ® can not be separated from industry partners, multi-experts and patients to promote.
    clinical data on patients with Chinese MPSII, particularly in overseas clinical trials at GC Pharma in Korea, has played an important role in driving the product's launch in China.
    'God Helps Self-Helpers', patients and experts from all walks of life are also driven by a spirit of forward-looking and relentless exploration that drives us to continue to work deep in the field of rare diseases.
    Dr. Xue Qun Beihai Kangcheng founder, chairman and chief executive officer "Beihai Kangcheng will fully promote the commercialization of rare diseases special effects drugs as a local-based new pharmaceutical creation company, Beihai Kangcheng has a focus on rare disease drug development platform, in the independent innovation at the same time, focus on the introduction of a series of foreign listed or in the late clinical stage of rare disease drugs, forming a product line collection, quickly bring effective drugs to patients with rare diseases."
    Beihai Kangcheng obtained the exclusive license agreement for the Greater China region of GC Pharma's drug Hunterase® ® in January 2019, and in July of the same year submitted an application for the listing of a new drug to China's State Drug Administration for formal acceptance, a rapid step towards commercialization of the rare disease sector.
    As a member and founding unit of the China Rare Disease Alliance, Beihai Kangcheng is not only committed to providing effective advanced drug treatment programs for patients with rare diseases, but also actively participated in promoting the construction of China's rare disease ecosystem, working with all parties to promote the improvement of relevant laws and regulations, and systematically addressing the issues of drug research and development and access to rare diseases.
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