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 today that Livmarli® (Maralixibat Chloride Oral Solution /邁芮倍®) has been granted an expanded label by the Taiwan Food and Drug Administration (TFDA). Livmarli received marketing approval in Taiwan for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS) aged one year or older in October 2023. This approval extends the use of Livmarli for the treatment of cholestatic pruritus in patients with ALGS to include those aged 2 months and older.

“We are delighted that Livmarli has received label expansion in Taiwan,” said Prof. Yao-Jong Yang, president of Taiwan Society of Pediatric Gastroenterology, Hepatology and Nutrition (TSPGHAN). “We believe this will allow diagnosed younger children to receive treatment sooner, alleviate symptoms, and improve their quality of life.”

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 Alagille Syndrome (ALGS)

Alagille syndrome (ALGS) is an autosomal dominant multisystem disorder which can lead to end-stage liver disease and death. Its incidence is between 1/30,000 and 1/50,000^[1]. It has been registered in National Rare Diseases Registry System of China (NRDRS). This disease is characterized by dysplasia of bile ducts and involvement of extrahepatic organs, such as the kidneys and eyes, as well as bones and the cardiovascular system. 100% of patients experience liver involvement ^{[2, 3]}, which often manifests as chronic cholestasis (slowed or stalled bile flow), usually in the neonatal period or within the first 3 months after birth. In addition to jaundice, skin xanthoma and hepatomegaly, patients will also experience severe pruritus ^[4], which can lead to skin disfigurement, emotional disorder, sleep deprivation and interruption of school learning, due to scratching in affected children ^[5]. It seriously affects the growth, development and quality of life of patients ^[6] and can lead to liver transplantation ^[7].

1.Kamath et al, JPGN 2018; 67: 148-156

2.Turnpenny PD, Ellard S. Eur J Hum Genet, 2012;20:251–57.

3.Saleh M, et al. Appl Clin Genet, 2016;9:75–82.

4.Elisofon SA, et al. J Pediatr Gastroenterol Nutr 2010; 51:759–765.

5.Elisofon et al. JPGN. 2010;51: 759-765.

6.Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527.

7.Kamath BM, et al. Hepatol Comms 2020; 4:387–398

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.

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

Contact:

China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com