"Kewang" Approved for Listing in China

The China Food and Drug Administration (SFDA) recently approved the new drug Kuvan, which was developed by Merck Serono, to be marketed in China. Kewang is also the first domestic drug that can be used to treat tetrahydrogen. Biopterin (BH4) deficiency drug.

Professor Yang Yanling, deputy head of the Neonatal Screening Unit of the Children's Health Branch of the Chinese Preventive Medicine Association and the Department of Pediatrics of the First Hospital of Peking University, said that BH4 deficiency is an autosomal-genetic disease that has been confirmed so far to be 5000 to 6,000 humans. One of the most rare diseases, BH4 deficiency, can be further diagnosed after screening for neonatal disease and detection of hyperphenylalaninemia (HPA). BH4 deficiency mainly causes damage to the human nervous system, causing children with mental retardation, epilepsy and other symptoms. Yang Yanling said: "BH4 deficiency is one of the few genetic diseases that can be controlled in a small number of cases. Kewang is allowed to be listed in China, making up for the gap in the clinical treatment of BH4 deficiency in China and bringing about the children and their families. new Hope."

Professor Shen Ming, a pediatrician at the China-Japan Friendship Hospital, said that it is estimated that approximately 20 to 200 million newborns in China may have BH4-deficiency ranging from about 100 to 200, that is, an average of 1 child with BH4-deficiency may be born every two days. . Since BH4 deficiency is a subtype of hyperphenylalaninemia, its incidence is even more rare. Dr. Kong Yuanyuan of Beijing Obstetrics and Gynecology Hospital said that drug treatment for BH4 deficiency is currently a more effective and safe treatment.

After the discovery of Kewang, in December 2007 and December 2008, it received rapid approval from the US FDA and the European Union EMA, and was granted the status of an orphan drug. Studies have shown that Kewang can effectively control the level of phenylalanine in the blood of children with BH4 deficiency, reduce the risk of sustained brain damage, and significantly improve the quality of life of children. In more than 600 clinical trials conducted abroad, the results were good.

Experts at the meeting called on the entire community to care about the survival and health rights of children with BH4-deficiency. As soon as possible, children with BH4-deficiency should be treated promptly to get rid of the physical and mental harm caused by the disease and contribute to the improvement of our population quality. It is understood that Shanghai Xinhua Hospital, China-Japan Friendship Hospital, Beijing University First Hospital and other hospitals have conducted differential diagnosis and treatment of BH4 deficiency. (Pan Feng Zhang Xiao)

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