Monday, July 15, 2024

China Approves CANbridge’s NDA for CAN108 to Treat Cholestatic Pruritus in PFIC

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The National Drug Administration (NDA) has received a submission based on data from the randomized Phase III MARCH PFIC study of LIVMARLI. The study incorporated 93 patients with various generic PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and patients with unidentified mutational status. The results showed patients treated with LIVMARLI experienced improvements in pruritus, serum bile acids, bilirubin, and growth, as measured by weight z-score, particularly in the cohort evaluating combined genetic subtypes.

LIVMARLI is touted as the first approved drug intended for the treatment of cholestatic pruritus associated with Alagille syndrome, in the US, Canada, Europe, and China. This marks a significant breakthrough in the field of genetic disease treatment across these regions. It is also noteworthy that LIVMARLI is currently being evaluated in a global Phase II EMBARK study in China for the treatment of biliary atresia (BA).

In 2021, an exclusive license agreement was signed between CANbridge and Mirum Pharmaceuticals for the development, manufacturing, and commercialization of LIVMARLI in Greater China. As a result of this agreement, CANbridge secured rights to develop, manufacture, and commercialize LIVMARLI for three indications, including Alagille syndrome, PFIC, and biliary atresia (BA), along with other indications, in Greater China.

Mirum has 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. This is a clear demonstration of the continued global efforts to combat this genetic disease and provide relief for affected patients.

James Xue, the founder, chairman, and CEO of CANbridge Pharmaceuticals, commented on the recent developments. He highlighted the previous approval of Livmarli for the treatment of patients with ALGS in mainland China, Hong Kong, and Taiwan and sees the PFIC as an opportunity for CANbridge to expand the label and maximize the potential of this product.

He also expressed anticipation for the formal approval of this indication in China, where he believes it holds the potential to provide much-needed relief to patients and their families. This optimism reflects the broader hopes of the medical community for improved treatment options for patients with these genetic conditions.

The recent developments signal positive progress in the treatment of PFIC. The submission to the NDA, based on the Phase III MARCH PFIC study of LIVMARLI, showed promising results with improvements in various symptoms. The approval of LIVMARLI for the treatment of cholestatic pruritus associated with Alagille syndrome is also a significant milestone. Furthermore, the exclusive license agreement between CANbridge and Mirum Pharmaceuticals broadens the potential for LIVMARLI’s development and commercialization in Greater China. Looking forward, the anticipation for further formal approval in China demonstrates the potential of LIVMARLI to offer relief for patients and their families affected by these conditions.

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