Approval

CANbridge Announces Approval Of CAN108 For Rare Liver Disease, Alagille Syndrome Under Early And Pilot Implementation Policy In Boao Lecheng Internati

March 01,2022 10:10 AM
- By Admin

CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company, announced that CAN108 (maralixibat), a treatment for Alagille syndrome (ALGS), has been approved under the Early and Pilot Implementation Policy in Boao Lecheng International Medical Tourism Pilot Zone, which will allow it to be imported and used as an urgently needed drug in the region.

Maralixibat was approved by the United States Food and Drug Administration (FDA) in September 2021 for the treatment of cholestatic pruritus in patients aged one year and older with ALGS. There are no approved drugs for the disease in China, where there is a large unmet need for treatment. The “Early and Pilot Implementation” Policy of Boao Lecheng International Medical Tourism Pilot Zone enables Chinese patients to access therapeutics that are available in other parts of the world, thereby improving the quality of life of patients, especially children.

CANbridge has the exclusive license to develop and commercialize CAN108 in Greater China for three rare liver disease indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA). The National Medical Products Administration (NMPA) has accepted a New Drug Application (NDA) for CAN108 for Alagille syndrome in China under priority review.

“Being able to offer this promising investigational treatment to Chinese patients soon after its US approval, is a realization of our core commitment to deliver complete patient solutions and part of why we founded the company,” said James Xue, Ph.D., CANbridge founder, chairman and CEO. “As the world takes note of another Rare Disease Day, we are pleased to be able to offer this specific kind of support to rare disease patients in China.”

Alagille syndrome (ALGS) is an autosomal dominant multisystem disorder, which can lead to end-stage liver disease and death. Its incidence is between 1/30000 and 1/50000 and it has been registered in National Rare Diseases Registry System of China (NRDRS). This disease is characterized by dysplasia of bile ducts and involvement of extrahepatic organs (such as cardiovascular system, bone, kidney, eyes) and appearance in children and adolescents. Among them, the incidence of liver involvement is 100%, which is often manifested as chronic cholestasis, usually in the neonatal period or within the first 3 months after birth. In addition to jaundice, skin xanthoma and hepatomegaly, patients will also experience severe pruritus, which can lead to skin disfigurement, emotional disorder, sleep deprivation and interruption of school learning due to scratching in affected children. It seriously affects the growth, development and quality of life of patients, and can lead to liver transplantation.

CAN108 is a barely absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the resultant liver injury and relieves pruritus. Maralixibat is the first and, currently, only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome.

In addition to ALGS, maralixibat is in the late stage of development for other cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA), both of which have been granted Breakthrough Therapy and Innovative Drugs for Rare Diseases designations by the FDA.

CANbridge Pharmaceuticals Inc. (“CANbridge,” stock code 1228.HK) is a China-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology.