Authors' objectives: Lopixibat is intended to be used for the treatment of progressive familial intrahepatic cholestasis (PFIC) in paediatric patients. If licensed, lopixibat will offer an additional treatment option for this patient group who currently have no therapies available that are specifically licensed for this condition. Lopixibat is an inhibitor of the sodium bile acid cotransporter that is in development for the treatment of cholestatic liver disorders in paediatric and adult patients. Lopixibat does not currently have Marketing Authorisation in the EU for any indication. PFIC is a rare disease. The precise prevalence of PFIC is unknown, but its estimated incidence varies between 1 in 50,000 and 1 in 100,000 live births worldwide. PFIC represents the cause of cholestasis in 10-15% of children with this finding, and is the indication for 10-15% of liver transplantations in children. Every year approximately 100 children or young people will receive a liver transplant in the UK. There are currently no approved therapies for PFIC in the USA or EU. Pharmaceutical and/or surgical treatments can reduce the effects and complications of the condition. Drugs used to improve bile flow and stimulate liver function include cholestyramine, ursodeoxycholic acid and rifampicin. Surgical intervention may include partial external biliary diversion, internal ileal exclusion and liver transplantation. Lopixibat is currently in a phase II clinical trial assessing its effect on fasting serum bile acids. The trial is expected to complete in October 2016.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.hsric.nihr.ac.uk/topics/lopixibat-for-progressive-familial-intrahepatic-cholestasis-in-paediatric-patients/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Research&Intelligence Centre (NIHR HSRIC)