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Please note that this product was withdrawn from the Union Register of orphan medicinal products in December 2019 on request of the Sponsor.

Key facts

Active substance
Disease / condition
Treatment of glucose transporter type-1 deficiency syndrome
Date of first decision
EU designation number

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 Netherlands B.V.
Evert van de Beekstraat 104
Schiphol Airport - The Base B
1118CN Schiphol
The Netherlands
Tel. +31620515586

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.

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