EU/3/12/1011: Orphan designation for the treatment of sickle cell disease



On 4 July 2012, orphan designation (EU/3/12/1011) was granted by the European Commission to Emmaus Medical Europe Limited, United Kingdom, for levoglutamide for the treatment of sickle cell disease.

The sponsorship was transferred to Emmaus Medical Europe Limited, Ireland, in May 2019.

Key facts

Active substance
Intended use
Treatment of sickle cell disease
Orphan designation status
EU designation number
Date of designation

Emmaus Medical Europe Limited
1 Anglesea Lane
Corrig Avenue
Dun Laoghaire 
Dublin A96 VK65
Tel. +353 1960 2030

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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