CANbridge Congratulates Mirum on EU Marketing Approval for Maralixibat/LIVMARLI® to Treat Cholestatic Pruritis in Rare Liver Disease, Alagille Syndrome
-Together with recent positive topline Phase 3 data in progressive familial intrahepatic cholestasis (PFIC), approval underscores maralixibat potential as promising new rare liver disease treatment
-CANbridge holds exclusive Greater China license to maralixibat (CAN108) in liver diseases, China part of global trial ongoing in biliary atresia (BA）
BEIJING and BURLINGTON, Mass., December 20, 2022—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, commented that the recent EU marketing authorization of maralixibat/LIVMARLI® for the treatment of cholestatic pruritus in patients with Alagille syndrome(ALGS) two months of age and older is a significant advancement in rare disease treatment options. Maralixibat is the first ALGS treatment approved in Europe. In addition, maralixibat developer, Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) recently reported positive topline data from its Phase 3 MARCH study evaluating the safety and efficacy of LIVMARLI® (maralixibat) in patients with progressive familial intrahepatic cholestasis (PFIC). CANbridge holds the exclusive right to develop,commercialize and, under certain conditions, manufacture maralixibat (CAN108) in Greater China for three liver diseases: biliary atresia (BA), ALGS and PFIC. CANbridge is overseeing the Phase 2 EMBARK study of CAN108 in BA in China. CAN108 has been approved to treat ALGS under the Early and Pilot Implementation Policy in the Boao Lecheng International Medical Tourism Pilot Zone, in China.
“The EU approval of maralixibat as the first treatment authorized in Europe for Alagille syndrome is the latest in a string of regulatory and clinical wins for maralixibat and underscores its potential as a promising new treatment for rare liver disease,” said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc. “We congratulate our partner, Mirum, as we continue to advance the development of CAN108 in Greater China,including a potential Hong Kong NDA filing, which may be submitted with regulatory approvals in two markets.”
About CAN108 (maralixibat)
CAN108 (maralixibat) is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the resultant liver injury and relieves pruritus (extreme itching). Maralixibat is the first and, currently, only medication approved in the US and EU to treat cholestatic pruritus associated with Alagille syndrome (ALGS).
In addition to ALGS, Maralixibat is under clinical development for the treatment of other cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA), and has been granted Breakthrough Therapy Designation by the FDA.
CANbridge acquired the exclusive right to develop, commercialize and, under certain conditions, manufacture CAN108 in Greater China from Mirum Pharmaceuticals, Inc. for ALGS, PFIC and BA. CAN108’s New Drug Application (NDA) for ALGS has been accepted and granted priority review by China’s National Medical Products Administration (NMPA).
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (HKEX:1228) global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.
For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.
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