On 28 July 2015, orphan designation (EU/3/15/1526) was granted by the European Commission to Ultragenyx UK Limited, United Kingdom, for triheptanoin for the treatment of carnitine palmitoyltransferase II 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 carnitine palmitoyltransferase II 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.