China-based rare disease specialist CANbridge Pharmaceuticals Inc. (HKG: 1228) has announced that a New Drug Application (NDA) for its maralixibat (CAN108) has been accepted for review by the National Medical Products Administration (NMPA) in China, with the added benefit of priority review status. This marks a significant step forward in providing treatment options for patients suffering from progressive familial intrahepatic cholestasis (PFIC), a rare genetic disease leading to progressive liver disease and ultimately liver failure.
PFIC: A Focus on Rare Disease Treatment
PFIC, included in the first batch of rare diseases released in China in May 2018, is a condition that requires specialized and targeted therapies. The NDA filing is based on the results of the Phase III MARCH PFIC study, the largest randomized trial conducted on PFIC, which covered a range of hereditary PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and undetermined mutation status.
Clinical Trial Success and Drug Efficacy
The Phase III MARCH PFIC study involved 93 patients, with those treated with maralixibat showing significant improvements in itching (P<0.0001), serum bile acids (P<0.0001), bilirubin (P=0.0471), and growth as measured by body weight Z-score (P=0.0391). These results underscore the drug’s potential in effectively managing the symptoms of PFIC.
Maralixibat: Mechanism and Regulatory Milestones
Originally discovered by US-based Mirum Pharmaceuticals, maralixibat is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reducing bile acid levels in the liver and serum, and relieving pruritus (itching) in patients with Alagille syndrome (ALGS) suffering from cholestatic pruritus. Mirum received US FDA approval for the drug in September 2021, marketed under the trade name Livmarli. CANbridge secured an exclusive license from Mirum to develop and commercialize maralixibat in Greater China in April 2021, focusing on three rare liver disease indications: ALGS, PFIC, and biliary atresia (BA). It was approved to treat pruritus due to cholestasis in patients with ALGS aged 1 year and above in China in June this year.-Fineline Info & Tech
