Maralixibat: First Approval

Maralixibat (Livmarli™) is an orally-administered, small-molecule ileal bile acid transporter (IBAT) inhibitor being developed by Mirum Pharmaceuticals for the treatment of rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and bi...

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Detalles Bibliográficos
Autor principal: Shirley, Matt
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
AdisInsight Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8748361/
https://www.ncbi.nlm.nih.gov/pubmed/34813049
http://dx.doi.org/10.1007/s40265-021-01649-0
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author Shirley, Matt
author_facet Shirley, Matt
author_sort Shirley, Matt
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description Maralixibat (Livmarli™) is an orally-administered, small-molecule ileal bile acid transporter (IBAT) inhibitor being developed by Mirum Pharmaceuticals for the treatment of rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia. Maralixibat received its first approval on 29 September 2021, in the USA, for use in the treatment of cholestatic pruritus in patients with ALGS 1 year of age and older. Maralixibat is also under regulatory review for ALGS in Europe, and clinical development for cholestatic liver disorders including ALGS in patients under 1 year of age, PFIC and biliary atresia is continuing in several other countries. This article summarises the milestones in the development of maralixibat leading to this first approval for ALGS. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40265-021-01649-0.
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spelling pubmed-87483612022-01-20 Maralixibat: First Approval Shirley, Matt Drugs AdisInsight Report Maralixibat (Livmarli™) is an orally-administered, small-molecule ileal bile acid transporter (IBAT) inhibitor being developed by Mirum Pharmaceuticals for the treatment of rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia. Maralixibat received its first approval on 29 September 2021, in the USA, for use in the treatment of cholestatic pruritus in patients with ALGS 1 year of age and older. Maralixibat is also under regulatory review for ALGS in Europe, and clinical development for cholestatic liver disorders including ALGS in patients under 1 year of age, PFIC and biliary atresia is continuing in several other countries. This article summarises the milestones in the development of maralixibat leading to this first approval for ALGS. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s40265-021-01649-0. Springer International Publishing 2021-11-23 2022 /pmc/articles/PMC8748361/ /pubmed/34813049 http://dx.doi.org/10.1007/s40265-021-01649-0 Text en © Springer Nature 2021, corrected publication 2021 https://creativecommons.org/licenses/by-nc/4.0/ Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle AdisInsight Report
Shirley, Matt
Maralixibat: First Approval
title Maralixibat: First Approval
title_full Maralixibat: First Approval
title_fullStr Maralixibat: First Approval
title_full_unstemmed Maralixibat: First Approval
title_short Maralixibat: First Approval
title_sort maralixibat: first approval
topic AdisInsight Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8748361/
https://www.ncbi.nlm.nih.gov/pubmed/34813049
http://dx.doi.org/10.1007/s40265-021-01649-0
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