LIVMARLI TM (maralixibat) oral solution was recently approved by the
'That the NMPA has accepted the NDA, which we anticipate will also receive priority review, for CAN108 for Alagille syndrome, our first candidate in rare liver disease, only 3 months after Maralixibat received FDA market approval, showcases our regulatory expertise and highlights the expanding list of indications we target,' said
About Alagille syndrome (ALGS
Alagille syndrome (ALGS) is a rare autosomal dominant disorder that affects multiple organs, including the liver, heart, bones, and eyes. Cholestatic liver disease is the most common form of it, and pruritus (severe itching) is the most severe clinical manifestation, in addition to jaundice, xanthomas, and hepatomegaly, which usually occur in infancy[1]. Pruritus may lead to skin mutilation, emotional disorder, interruption of sleep and school learning activities[2], seriously affecting children's growth, development, and quality of life[3]. Severe cholestatic pruritus is also an indication for liver transplantation[4]. According to
About Maralixibat
LIVMARLI (Maralixibat) is a barely 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. LIVMARLI TM is the first and, currently, only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome.
Maralixibat is an oral drug that, in addition to ALGS, is under advanced 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 and Innovative Drugs for Rare Diseases designations by the FDA.
About
CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology.
These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) as well as glioblastoma multiforme (GBM).
CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to,
Contact:
E: ir@canbridgepharma.com
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E: deanne@planetcommunications.nyc
T: 917.837.5866
[1]
[2] Elisofon et al. JPGN. 2010;51: 759-765
[3] Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527
[4]Guidelines for the Management of Cholestatic Liver Diseases (2021)
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