EU/3/13/1232: Orphan designation for the treatment of epidermolysis bullosa



Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2020 on request of the Sponsor.

On 16 January 2014, orphan designation (EU/3/13/1232) was granted by the European Commission to ORS Oxford Ltd, United Kingdom, for allantoin for the treatment of epidermolysis bullosa.

The sponsorship was transferred as follows:
•    to Scioderm Limited, Ireland, in October 2014;
•    to Amicus Therapeutics UK Ltd, United Kingdom, February 2016;
•    and finally to Amicus Therapeutics Europe Limited, Ireland, in March 2019.

Key facts

Active substance
Intended use
Treatment of epidermolysis bullosa
Orphan designation status
EU designation number
Date of designation

Amicus Therapeutics Europe Limited
Block 1, Blanchardstown Corporate Park
Ballycoolin Road
Dublin 15
D15 AKK1
Tel. +353 1 588 6850

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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