CANbridge Pharmaceuticals recently announced via press release that the Taiwan Food and Drug Administration (TFDA) has accepted their New Drug Application (NDA)/Orphan Drug Registration (ODR) for CAN108. Marketed as LIVMARLI, CAN108 is a treatment for Alagille syndrome. With this approval, Alagille syndrome patients in Taiwan now have a new treatment option.

About Alagille Syndrome

First up, let’s dive in to what this rare disease is. Alagille syndrome is a rare, genetic disorder that impacts multiple systems throughout the body, including the liver, kidneys, skeleton, eyes, and heart. The effects on these systems can vary widely throughout affected individuals, but liver disease and heart abnormalities are typically seen. Other symptoms include failure to thrive, jaundice, abnormalities of the back bone, distinctive facial features, pruritus, heart defects, cholestasis, heart murmurs, and pale and loose stools. These symptoms are the result of a mutated JAG1 or NOTCH2 gene. Both are usually inherited in an autosomal dominant pattern, but they can be sporadic mutations as well. There is currently no cure for Alagille syndrome; treatment is symptomatic and supportive.

Update on CAN108

In Taiwan, CAN108 is indicated for the treatment of cholestatic pruritus in those with Alagille syndrome who are at least one year old. However, this is not the first approval that CAN108 (also known as LIVMARLI or maralixibat) has received – in the United States, the FDA has granted approval to CAN108 for the same indication that Taiwan’s regulatory agency did. China’s National Medical Products Administration has granted the same approval.

While the approvals for this drug are all in regard to cholestatic pruritus caused by Alagille syndrome, an agreement between CANbridge and Miurm Pharmaceuticals states that it can be commercialized for three indications: Alagille syndrome, biliary atresia (BA), and progressive familial intrahepatic cholestasis (PFIC) within China.