China’s National Medical Products Administration (NMPA) accepted the New Drug Application (NDA) for CAN108 (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 1 year and above.

“That the NMPA has accepted the NDA, which we anticipate will also receive priority review, for CAN108 for [ALGS], our first candidate in rare liver disease, only 3 months after Maralixibat received [US Food and Drug Administration] market approval, showcases our regulatory expertise and highlights the expanding list of indications we target,” James Xue, PhD, founder and chief executive officer of CANbridge said in a press release.

“We look forward to developing this treatment in China, where it could bring much-needed relief to patients and their families.”


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ALGS is a rare genetic disease caused by the disruption of the Notch signaling pathway leading to intrahepatic bile duct paucity. This means that bile acids cannot be drained from the liver efficiently enough causing chronic cholestasis.

The symptoms of the disease include pruritus, jaundice, xanthoma, dark urine, light, loose, and greasy stools, hepatomegaly, and splenomegaly. It is estimated that there are 7,400 people living with ALGS in China and 68,000 living with the condition worldwide. 

Read more about ALGS symptoms

Maralixibat is an apical sodium-dependent bile acid transporter inhibitor. This inhibition leads to more bile acid being excreted in the feces, thereby reducing systemic bile acid levels. The treatment could, therefore, reduce liver damage caused by the build-up of bile acid and improve liver function in patients with ALGS.

The treatment was recently approved by the US Food and Drug Administration for the treatment of cholestatic pruritus in patients with ALGS 1 year and older making it the first and only medication that the agency approved to treat ALGS. 

Clinical trials are underway to test the safety and efficacy of maralixibat in other cholestatic liver diseases including progressive familial intrahepatic cholestasis and biliary atresia.

Reference

CANbridge pharmaceuticals CAN108 New Drug Application (NDA), for Alagille syndrome, accepted by China’s National Medical Products Administration. News release. CANbridge; January 17, 2022.