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 and
  • to FGK Representative Service GmbH, Germany, in April 2020.

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)
Disease / condition
Treatment of progressive familial intrahepatic cholestasis
Date of first decision
EU designation number

Review of designation

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

Sponsor's contact details

FGK Representative Service GmbH
Heimeranstrasse 35
80339 Munich

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.

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