EU/3/15/1526: Orphan designation for the treatment of carnitine palmitoyltransferase II deficiency
Triheptanoin
Table of contents
Overview
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.
The sponsor’s address was updated in November 2020.
Key facts
Active substance |
Triheptanoin
|
Intended use |
Treatment of carnitine palmitoyltransferase II deficiency
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/15/1526
|
Date of designation |
28/07/2015
|
Sponsor |
Ultragenyx Germany GmbH |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: