On 21 May 2015, orphan designation (EU/3/15/1495) was granted by the European Commission to Pharma Gateway AB, Sweden, for triheptanoin for the treatment of glucose transporter type-1 deficiency syndrome.
The sponsorship was transferred to Ultragenyx UK Limited, United Kingdom, in August 2015.
The sponsorship was transferred to Ultragenyx Netherlands B.V., The Netherlands, in October 2018.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2019 on request of the Sponsor.
|Disease / condition||
Treatment of glucose transporter type-1 deficiency syndrome
|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.