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.
|Disease / condition||
Treatment of very long-chain acyl-CoA dehydrogenase deficiency
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.