BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals, Inc. (“CANbridge”, stock code 1228.HK), a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative therapies, announced that CAN108 (maralixibat), a treatment for Alagille syndrome (ALGS), has been approved by the Hainan Medical Products Administration, which will allow it to be imported and used in Boao Lecheng International Medical Tourism Pilot Zone as an urgently needed drug.

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.”

About Alagille Syndrome (ALGS)

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 [1] 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% [2, 3], 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 [4], which can lead to skin disfigurement, emotional disorder, sleep deprivation and interruption of school learning, due to scratching in affected children [5]. It seriously affects the growth, development and quality of life of patients [6] and can lead to liver transplantation [7].

About CAN108 (maralixibat)

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.

Maralixibat is an oral drug that, in addition to ALGS, is under advanced clinical development for the treatment of other cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA), and has been granted Breakthrough Therapy and Innovative Drugs for Rare Diseases designations by the FDA.

About CANbridge Pharmaceuticals Inc.

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.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology. These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass) and LogicBio.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

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3. Saleh M, et al. Appl Clin Genet, 2016;9:75–82.

4. Elisofon SA, et al. J Pediatr Gastroenterol Nutr 2010; 51:759–765.

5. Elisofon et al. JPGN. 2010;51: 759-765.

6. Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527.

7. Kamath BM, et al. Hepatol Comms 2020; 4:387–398.