EU/3/15/1526: Orphan designation for the treatment of carnitine palmitoyltransferase II deficiency



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
Intended use
Treatment of carnitine palmitoyltransferase II deficiency
Orphan designation status
EU designation number
Date of designation

Ultragenyx Germany GmbH
Rahel-Hirsch-Strasse 10
10557 Berlin
Tel. +4930590083651

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

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:

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