EU/3/13/1216

About

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
16/01/2014
Outcome
Positive
EU designation number
EU/3/13/1216

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
Schwanthalerhoehe-Laim
80339 Munich
Germany
E-mail: info@imv-inc.com

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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