Overview
On 13 November 2020, orphan designation EU/3/20/2360 was granted by the European Commission to Ultragenyx Germany GmbH, Germany, for triheptanoin for the treatment of carnitine palmitoyltransferase I deficiency.
Key facts
Active substance |
Triheptanoin
|
Intended use |
Treatment of carnitine palmitoyltransferase I deficiency
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/20/2360
|
Date of designation |
13/11/2020
|
Sponsor |
Ultragenyx Germany GmbH |
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: