EU/3/20/2360: Orphan designation for the treatment of carnitine palmitoyltransferase I deficiency

Triheptanoin

Table of contents

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
Rahel-Hirsch-Strasse 10
Moabit
10557 Berlin
Germany
Tel: +493020659217
E-mail: inquiry@ultragenyx.com

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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