CANbridge Pharmaceuticals Inc. announced that the National Medical Products Administration of China (NMPA) has accepted the New Drug Application (NDA) for CAN108 (Maralixibat Chloride Oral Solution /???®/LIVMARLI®) for the treatment of cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. PFIC has been included in the "First National List of Rare Diseases" in China since May 2018.

The application has been granted priority review. The submission is based on data from the MARCH PFIC Phase 3 study of LIVMARLI. MARCH PFIC is the larger randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status.

In the study, LIVMARLI-treated patients had statistically significant improvements in pruritus (p< 0.0001), serum bile acids (p<0.0001), bilirubin (p=0.0471), and growth as measured by weight z-score (p=0.0391), in the cohort evaluating combined genetic subtypes. LIVMARLI is the first approved drug to treat cholestatic pruritus associated with Alagille syndrome in the US, Canada, Europe and China. CANbridge and Mirum Pharmaceuticals Inc. (Mirum) signed an exclusive license agreement for the development, manufacturing and commercialization of LIVMARLI in Greater China in 2021.

Mirum has also submitted a supplemental NDA to the U.S. Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients three months of age and older with PFIC, as well as a marketing authorization variation to the European Medicines Agency (EMA) for the treatment of PFIC. Under the terms of the agreement, CANbridge has the right to develop manufacture and commercialize LIVMARLI for three indications: Alagille syndrome (ALGS), PFIC and biliary atresia (BA), along with select other indications, in Greater China. LIVMARLI is currently being evaluated in an ongoing Global Phase 2 study in China known as EMBARK for the treatment of BA.