Alagille syndrome is an autosomal genetic abnormality that causes cholestasis, congenital cardiovascular disease, abnormal bone structure, corneal abnormalities, and a distinctive appearance. Sick children usually develop jaundice within two or three months after birth. Due to the inability to excrete bile, blood tests reveal that the bilirubin and bile acids in the body are higher than normal. 50% to 90% of sick children will suffer from growth retardation and severe itching, which can easily lead to sleep and mood disorders.
Alajerry Syndrome is not uncommon, studies have shown that 1 in every 30,000 to 50,000 babies suffers from it. Therefore, if parents find that the jaundice of the newborn has not subsided after the full moon, they need to seek medical treatment as soon as possible. In recent years, domestic attention has been paid to Alajri syndrome, which has been registered in the National Rare Disease Registration System of China.
On February 20, 2022, Beihai Kangcheng, a domestic rare disease pharmaceutical company “Unicorn”, passed the approval of Livmarli Oral Liquid, a drug for the treatment of Alajelli syndrome, which is allowed to be imported as a clinically urgently needed drug in Lecheng International Medical Tourism Pilot Zone, Boao, Hainan This is a major breakthrough in the experimental treatment of Alajri syndrome in China. Livmarli oral solution is used for the treatment of cholestatic pruritus in patients with Alajri syndrome. There is no other approved treatment drug for this indication in China. Beihai Kangcheng’s brand-new “first-in-first-out” plan allows Chinese patients in Alajri to enjoy a disease treatment plan that is synchronized with the world. At present, the State Drug Administration has accepted the new drug marketing authorization application of Livmarli Oral Liquid for the treatment of Alajri syndrome in China, and it is in the priority review stage. At the same time, Beihai Kangcheng is committed to the research and development of rare disease biological agents and small molecule products and candidate products such as paroxysmal nocturnal hemoglobinuria (PNH) and glioblastoma (GBM), hoping to improve the quality of life of patients, especially children .
Beihai Kangcheng scientific research team
