EU/3/20/2302
Table of contents
About
On 27 July 2020, orphan designation EU/3/20/2302 was granted by the European Commission to Ultragenyx Germany GmbH, Germany, for triheptanoin for the treatment of carnitine-acylcarnitine translocase deficiency.
Key facts
Active substance |
Triheptanoin
|
Disease / condition |
Treatment of carnitine-acylcarnitine translocase deficiency
|
Date of first decision |
27/07/2020
|
Outcome |
Positive
|
EU designation number |
EU/3/20/2302
|
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
Rahel-Hirsch-Strasse 10
Moabit
10557 Berlin
Germany
Tel. +4930590083651
E-mail: medinfo@ultragenyx.com
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.