EU/3/12/1082: Orphan designation for the treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency



Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in February 2016 on request of the Sponsor.

On 6 December 2012, orphan designation (EU/3/12/1082) was granted by the European Commission to B. Braun Melsungen AG, Germany, for triheptanoin for the treatment of long-chain 3-hydroxyacyl-CoA-dehydrogenase deficiency.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Key facts

Active substance
Intended use
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency
Orphan designation status
EU designation number
Date of designation
B. Braun Melsungen AG
Carl-Braun-Strasse 1
34212 Melsungen
Tel. +49 56 61 710
Fax +49 56 61 71 2950

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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