Enterohepatic circulation

CANbridge Announces Encouraging Development in Maralixibat/LIVMARLI in Rare Liver Disease

Retrieved on: 
Thursday, October 27, 2022

PFIC is included in the 121 Rare Disease List published in 2018 by Chinese authorities.

Key Points: 
  • PFIC is included in the 121 Rare Disease List published in 2018 by Chinese authorities.
  • We congratulate Mirum on the trial success and look forward to continuing to work together to bring potential new treatments to rare liver disease patients in Greater China, said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.
  • 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 and rare oncology.
  • CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 11 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential.

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

Retrieved on: 
Tuesday, March 29, 2022

CANbridge recently received an acceptance letter for a CAN108 NDA the same indication from Chinas National Medical Products Administration.

Key Points: 
  • CANbridge recently received an acceptance letter for a CAN108 NDA the same indication from Chinas 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.
  • As our first therapeutic under development for rare liver disease, CAN108 deepens our rare disease portfolio and highlights the swiftness of our regulatory progress.
  • Alagille syndrome (ALGS) is a rare autosomal dominant disorder that affects multiple organs, including the liver, heart, bones and eyes.

CANbridge Announces Approval of CAN108 for Rare Liver Disease, Alagille Syndrome, Under the Early and Pilot Implementation Policy in Boao Lecheng International Medical Tourism Pilot Zone

Retrieved on: 
Friday, February 25, 2022

The Early and Pilot Implementation Policy of Boao Lecheng International Medical Tourism Pilot Zone enables Chinese patients to access therapeutics that are available in other parts of the world, thereby improving the quality of life of patients, especially children.

Key Points: 
  • The Early and Pilot Implementation Policy of Boao Lecheng International Medical Tourism Pilot Zone enables Chinese patients to access therapeutics that are available in other parts of the world, thereby improving the quality of life of patients, especially children.
  • CANbridge has the exclusive license to develop and commercialize CAN108 in Greater China for three rare liver disease indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA).
  • The National Medical Products Administration (NMPA) has accepted a New Drug Application (NDA) for CAN108 for Alagille syndrome in China under priority review.
  • Alagille syndrome (ALGS) is an autosomal dominant multisystem disorder which can lead to end-stage liver disease and death.

CANbridge Pharmaceuticals CAN108 New Drug Application (NDA) for Alagille Syndrome Accepted by China’s National Medical Products Administration

Retrieved on: 
Tuesday, January 18, 2022

CANbridge anticipates that the application will be granted priority review, upon the close of the publicity period of the priority review process, on January 17th.

Key Points: 
  • CANbridge anticipates that the application will be granted priority review, upon the close of the publicity period of the priority review process, on January 17th.
  • CANbridge and Mirum Pharmaceuticals signed an exclusive license agreement for the development and commercialization 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.
  • Alagille syndrome (ALGS) is a rare autosomal dominant disorder that affects multiple organs, including the liver, heart, bones and eyes.

Mirum Pharmaceuticals Announces Data Presented During AASLD Highlighting Durable Improvements in Pruritus and Quality of Life in Children with Alagille Syndrome Treated with Maralixibat

Retrieved on: 
Sunday, November 15, 2020

Maralixibat, an apical sodium bile acid transporter (ASBT) inhibitor, has previously been shown to interrupt the enterohepatic circulation of bile acids, reducing pruritus .

Key Points: 
  • Maralixibat, an apical sodium bile acid transporter (ASBT) inhibitor, has previously been shown to interrupt the enterohepatic circulation of bile acids, reducing pruritus .
  • Maralixibat treatment improved quality of life and led to improved growth parameters.
  • To view the presentation and the complete data, please visit the AASLD section within the Events page on Mirums website.
  • Children receiving maralixibat in the studies also demonstrated improvements in biomarkers of disease, including reductions in cholesterol and bile acid levels.

Mirum Pharmaceuticals Presents Maralixibat Five-Year Transplant-Free Survival Data for Patients With PFIC2 at Digital International Liver Congress 2020

Retrieved on: 
Saturday, August 29, 2020

The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.

Key Points: 
  • The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.
  • Data presented today demonstrate that, similar to the findings of NAPPED, patients exhibiting sBA control with pharmacological interruption of the enterohepatic circulation with maralixibat have significantly improved transplant-free survival (p=0.0006).
  • INDIGO included 19 PFIC2 patients with non-truncating BSEP mutations who received maralixibat 280 g/kg once or twice daily.
  • In a Phase 2 PFIC study , a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat.