EU/3/06/368

About

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;
  • and finally to Amicus Therapeutics UK Ltd, United Kingdom, in March 2014.

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.

Key facts

Active substance
1-Deoxygalactonojirimycin hydrochloride
Disease / condition
Treatment of Fabry disease
Date of decision
22/05/2006
Outcome
Positive
Orphan decision number
EU/3/06/368

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.

Sponsor's contact details

Amicus Therapeutics UK Ltd
Phoenix House, Oxford Road
Tatling End
Gerrards Cross
Buckinghamshire SL9 7AP
United Kingdom
Tel. + 44 (0)1844 211669
Fax + 44 (0)1844 211081
E-mail: info@amicustherapeutics.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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