EU/3/13/1214: Orphan designation for the treatment of Alagille syndrome

(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)


On 18 December 2013, orphan designation (EU/3/13/1214) 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 Alagille syndrome.

The medicinal product has been authorised in the EU as Livmarli since 09 December 2022.

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 Alagille syndrome
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 reviewed the orphan designation of Livmarli at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the PDF icon orphan medicine assessment report .

Update history

September 2021The sponsorship was transferred to Mirum Pharmaceuticals International B.V., Netherlands.
December 2019The sponsorship was transferred to Granzer Regulatory Consulting & Services, Germany.
March 2019The sponsorship was transferred to SFL Regulatory Services GmbH, Austria.
September 2016The sponsorship was transferred to Shire Pharmaceuticals Ireland Limited, Ireland.

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:

Related content

How useful was this page?

Add your rating