The National Medical Products Administration (NMPA) of China has accepted the new drug application (NDA) submitted by CANbridge Pharmaceuticals for CAN108 (Maralixibat Chloride Oral Solution/ LIVMARLI) for the treatment of cholestatic pruritus in patients aged two months and above with progressive familial intrahepatic cholestasis (PFIC).
The NDA has been submitted based data from the randomised Phase III MARCH PFIC study of LIVMARLI.
A total of 93 patients with various generic PFIC subtypes including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status were enrolled into the study.
Patients treated with LIVMARLI showed 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 claimed to be the first approved drug intended for the treatment of cholestatic pruritus associated with Alagille syndrome, in the US, Canada, Europe and China.
In 2021, CANbridge and Mirum Pharmaceuticals signed an exclusive license agreement for the development, manufacturing and commercialisation of LIVMARLI in Greater China.
As part of the agreement, CANbridge gained rights to develop, manufacture and commercialise LIVMARLI for three indications including Alagille syndrome, PFIC and biliary atresia (BA), along with other indications, in Greater China.
Mirum also submitted a supplemental NDA of CAN108 to the US Food and Drug Administration (FDA) to seek approval for CAN108 for treating cholestatic pruritus in patients aged three months and above with PFIC.
LIVMARLI is also being assessed in a global Phase II EMBARK study in China for the treatment of BA.
CANbridge Pharmaceuticals founder, chairman and CEO James Xue said: “With the approval of Livmarli for the treatment of patients with ALGS in mainland China, Hong Kong and Taiwan already received earlier this year, PFIC represents the opportunity for CANbridge to expand the label and maximize the potential of this product.
“We eagerly anticipate the formal approval of this indication in China, where it holds the potential to provide much-needed relief to patients and their families.”