EU/3/06/368: Orphan designation for the treatment of Fabry disease
Table of contents
On 22 May 2006, orphan designation (EU/3/06/368) was granted by the European Commission to Amicus Therapeutics UK Limited, United Kingdom, for 1-deoxygalactonojirimycin hydrochloride for the treatment of Fabry disease.
The sponsorship was transferred as follows:
- to Shire Pharmaceutical Development Limited, United Kingdom, in June 2008;
- to Amicus Therapeutics UK Ltd, United Kingdom, in February 2010;
- to Glaxo Group Limited, United Kingdom, in June 2011;
- to Amicus Therapeutics UK Ltd, United Kingdom, in March 2014;
- and finally to Amicus Therapeutics Europe Limited, Ireland, in March 2019.
Update: 1-deoxygalactonojirimycin hydrochloride has been authorised in the EU as Galafold since 26 May 2016.
More information on Galafold can be found in the European public assessment report (EPAR) on the Agency's website.
Treatment of Fabry disease
|Orphan designation status||
|EU designation number||
|Date of designation||
Amicus Therapeutics Europe Limited
Review of designation
On 8 April 2016, the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/06/368 for Galafold (migalastat1) as an orphan medicinal product for the treatment of Fabry disease. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with Fabry disease. The COMP recommended that the orphan designation of the medicine be maintained2.
1Previously known as 1-deoxygalactonojirimycin hydrochloride.
2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.
Documents related to this orphan designation evaluation
Recommendation for maintenance of orphan designation at the time of marketing authorisation: Galafold (migalastat) for the treatment of Fabry disease (PDF/83.66 KB)
First published: 10/06/2016
Last updated: 10/06/2016
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: