SUZHOU —Dec 18, 2024 — CANbridge Pharmaceuticals, Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases, announced the marketing approval of Livmarli® (Maralixibat Chloride Oral Solution /邁芮倍®)  by the Taiwan Food and Drug Administration (TFDA) for Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis (PFIC) patients aged 3 months and older.

“We are delighted that Livmarli is now available to PFIC patients in Taiwan,” said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals Inc. “We are committed to making Livmarli accessible to all PFIC patients throughout the greater China region. Armed with the first and only approved therapy for PFIC, we will work hard to realize the full potential of Livmarli for  addressing the unmet medical needs of PFIC patients in Taiwan.”

About LIVMARLI® (Maralixibat Chloride Oral Solution /迈芮倍®)

LIVMARLI® (Maralixibat Chloride Oral Solution /迈芮倍®) 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). LIVMARLI is the first, and only medication approved in China, the US and EU (2 months and older) to treat cholestatic pruritus associated with Alagille syndrome (ALGS) aged 3 months and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older.

LIVMARLI has been granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC by the FDA.

About PFIC

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in affected individuals. Signs and symptoms of PFIC typically begin in infancy. Patients experience severe itching, jaundice, failure to grow at the expected rate (failure to thrive), and an increasing inability of the liver to function (liver failure). The disease is estimated to affect one in every 50,000 to 100,000 births in the United States and Europe.  Six types of PFIC have been genetically identified, all of which are similarly characterized by impaired bile flow and progressive liver disease.

About CANbridge Pharmaceuticals Inc.  

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 9 assets, targeting prevalent rare disease 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. 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), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, 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.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare diseases. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 9 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome (Mucopolysaccharidosis type II) and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. 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), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Biopharma, Mirum Pharma, WuXi Biologics, Privus Biologics, 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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CANbridge Pharmaceuticals Inc.

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