On 19 June 2015, orphan designation (EU/3/15/1508) was granted by the European Commission to Ultragenyx UK Limited, United Kingdom, for triheptanoin for the treatment of very long-chain acyl-CoA dehydrogenase deficiency.

The sponsorship was transferred first to Ultragenyx Netherlands B.V., the Netherlands, in February 2019 and subsequently to Ultragenyx Germany GmbH, Germany, in February 2020.

Key facts

Active substance
Medicine name
Disease / condition
Treatment of very long-chain acyl-CoA dehydrogenase deficiency
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

Ultragenyx Germany GmbH
Friedrichstrasse 191
10117 Berlin
Tel. +49 30 2065 9217

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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