EU/3/20/2360

Table of contents

About

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
Disease / condition
Treatment of carnitine palmitoyltransferase I deficiency
Date of first decision
13/11/2020
Outcome
Positive
EU designation number
EU/3/20/2360

Sponsor's contact details

Ultragenyx Germany GmbH
Rahel-Hirsch-Strasse 10
Moabit
10557 Berlin
Germany
Tel: +493020659217
E-mail: inquiry@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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