CANbridge Pharmaceuticals Inc. announced the marketing approval of CAN108 (Maralixibat Chloride Oral Solution /LIVMARLI®?) by the Taiwan Food and Drug Administration (TFDA). LIVMARLI is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, and the first and only treatment approved medication in Taiwan for the treatment of cholestatic pruritus (itching caused by slowed or stalled bile flow) in patients with Alagille syndrome (ALGS) aged one year or older. ALGS is a rare genetic disorder that can lead to end-stage liver disease and death.

ALGS is characterized by paucity of the bile ducts, which causes cholestasis, and involvement of extrahepatic organs, such as the kidneys, eyes, as well as bones and the cardiovascular system. Cholestatic pruritus is the most burdensome symptom in ALGS, greatly reducing quality of life. LIVMARLI has recently been approved in Hong Kong, mainland China and Canada.

It has also been approved in the United States for patients with ALGS aged three months and older, and in Europe for patients with ALGS aged two months and older. LIVMARLI is the first approved drug to treat cholestatic prurus associated with Alagille syndrome in the US, Europe, and China. LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases, including BA.

LIVMARLI has been granted Breakthrough Therapy designations for ALGS and PFIC type 2, and orphan designations for ALGS, PFIC and BA, by the FDA. Alagille syndrome (ALGS) is an autosomal dominant, multisystem disorder which can lead to end-stage Liver disease and death. The birth prevalence of ALGS is between 1/30,000 and 1/50,000, and it has been registered in the National Rare Diseases Registry System of China (NRDRS).

ALGS has been included in the "Second National List of Rare Diseases" in China in September 2023. The disease is characterized by a paucity of bile ducts and involvement of extrahepatics organs, such as the kidneys and eyes, as well as bone and the cardiovascular system. All patients experience liver involvement, which often manifests as chronic cholestasis (slowed or stalled bile flow), usually in the neonatal period or within the first 3 months after birth.

In addition to jaundice, the liver and serum, and the cardiovascular system.