EU/3/20/2302

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.

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