EU/3/14/1310: Orphan designation for the treatment of Fabry disease


This medicine is now known as venglustat.

On 22 August 2014, orphan designation (EU/3/14/1310) was granted by the European Commission to Genzyme Europe BV, the Netherlands, for (3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate for the treatment of Fabry disease.

The sponsor’s address was updated in December 2018.

Key facts

Intended use
Treatment of Fabry disease
Orphan designation status
EU designation number
Date of designation

Sanofi B.V.
Paasheuvelweg 25
1105 BP Amsterdam

Review of designation

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

Update history

July 2023The sponsor’s name was changed from Genzyme Europe B.V. to Sanofi B.V. in July 2023.

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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