BEIJING - CANbridge Pharmaceuticals Inc. (HKEX: 1228), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced today that the National Medical Products Administration of China (NMPA) has accepted the New Drug Application (NDA) for CAN108 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 largest 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

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