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)


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
EU designation number
Date of designation

Mirum Pharmaceuticals International B.V.
Kingsfordweg 151
1043 GR Amsterdam
Tel: +31 204919191

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

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:

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