EU/3/13/1217

About

On 16 January 2014, orphan designation (EU/3/13/1217) 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 primary sclerosing cholangitis.

The sponsorship was transferred to Shire Pharmaceuticals Ireland Limited, Ireland, in September 2016 and subsequently to SFL Regulatory Services GmbH, Austria, in March 2019.

The sponsorship was transferred to Granzer Regulatory Consulting & Services, Germany in December 2019.

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
Disease / condition
Treatment of primary sclerosing cholangitis
Date of first decision
16/01/2014
Outcome
Positive
EU designation number
EU/3/13/1217

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

Granzer Regulatory Consulting & Services
Kistlerhofstrasse 172c
81379 Munich
Germany
Tel. +49 2217 401339
E-mail: medinfo@mirumpharma.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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