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CANbridge Pharmaceuticals CAN108 New Drug Application/Orphan Drug Registration (NDA/ORD) for Alagille Syndrome Accepted by the Taiwan Food and Drug Administration

2022年03月29日 AM09:00
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BEIJING & CAMBRIDGE, Mass.

CANbridge Pharmaceuticals Inc., (“CANbridge,” stock code 1228.HK) a leading China-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the Taiwan Food and Drug Administration (TFDA) has accepted the New Drug Application/Orphan Drug Registration (NDA/ODR) for CAN108 (maralixibat oral solution (LIVMARLI TM)) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. LIVMARLI TM (maralixibat) oral solution was recently approved by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. CANbridge recently received an acceptance letter for a CAN108 NDA the same indication from China’s National Medical Products Administration.

CANbridge and Mirum Pharmaceuticals signed an exclusive license agreement for the development, commercialization and manufacture, under certain conditions, of maralixibat (CAN108) in Greater China last year. Under the terms of the agreement, CANbridge has the right to develop and commercialize CAN108 for three indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) in Greater China.

“We are pleased that Taiwan’s FDA accepted the NDA for CAN108 for Alagille syndrome so soon after approval of this life-changing drug in the US, reflecting our goal to shorten the runway between approval of rare disease therapeutics in other markets, and developing them in Asia,” said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. “As our first therapeutic under development for rare liver disease, CAN108 deepens our rare disease portfolio and highlights the swiftness of our regulatory progress.”

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 infancyi. Pruritus may lead to skin mutilation, emotional disorder, interruption of sleep and school learning activitiesii, seriously affecting children’s growth, development and quality of lifeiii. Severe cholestatic pruritus is also an indication for liver transplantationiv. According to Frost & Sullivan, there were 68,000 ALGS patients in the world, and 7400 patients in China in 2020.

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 Innovative Drugs for Rare Diseases designation. ALGS and PFIC have been granted Breakthrough Therapy by the FDA. Mirum Pharmaceuticals Inc. is conducting a Phase 3 clinical trial of maralixibat in PFIC in North America, Europe, Asia, and South America. A global maralixibat phase 2 clinical trial (EMBARK) in BA is also underway, for which CANbridge is managing the China sites.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (“CANbridge,” stock code: 1228.HK) is a China-based global rare disease-focused biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies.

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, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass), the University of Washington School of Medicine, LogicBio and Scriptr.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

 


i Binita M. Kamath et al. JPGN. 2018;67: 148-156
ii Elisofon et al. JPGN. 2010;51: 759-765
iii Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527
ivGuidelines for the Management of Cholestatic Liver Diseases (2021)

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220328005731/en/

CONTACT

U.S. Investor Relations:

Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com

Media:

Deanne Eagle

Planet Communications

deanne@planetcommunications.nyc

917.837.5866

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