EU/3/03/154: Orphan designation for the treatment of sickle cell syndrome


Table of contents


Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2017 at the end of the 10-year period of market exclusivity.

On 9 July 2003, orphan designation (EU/3/03/154) was granted by the European Commission to OTL Pharma, France, for hydroxyurea for the treatment of sickle cell syndrome. The sponsorship was transferred to Addmedica SAS, France, in October 2006.

Hydroxyurea has been authorised in the EU as Siklos since 29 June 2007.

Key facts

Active substance
Medicine name
Intended use
Treatment of sickle cell syndrome
Orphan designation status
EU designation number
Date of designation
Addmedica SAS
101 rue Saint Lazare
75009 Paris
Telephone: + 33 1 72 69 01 86
Telefax: + 33 1 73 72 94 13

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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