EU/3/13/1216: Orphan designation for the treatment of progressive familial intrahepatic cholestasis
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride)
Table of contents
Overview
This medicine is now known as maralixibat chloride.
On 16 January 2014, orphan designation (EU/3/13/1216) was granted by the European Commission to Lumena Pharma UK Limited, United Kingdom, for (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride for the treatment of progressive familial intrahepatic cholestasis.
The sponsorship was transferred as follows:
- to Shire Pharmaceuticals Ireland Limited, Ireland, in September 2016,
- to SFL Regulatory Services GmbH, Austria, in March 2019,
- to Granzer Regulatory Consulting & Services, Germany, in December 2019,
- to FGK Representative Service GmbH, Germany, in April 2020,
- to Mirum Pharmaceuticals International B.V. in November 2021.
Key facts
Active substance |
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride)
|
Intended use |
Treatment of progressive familial intrahepatic cholestasis
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/13/1216
|
Date of designation |
16/01/2014
|
Sponsor |
Mirum Pharmaceuticals International B.V. |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: