Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in July 2017 at the end of the 10-year period of market exclusivity.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.
Treatment of sickle cell syndrome
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Documents related to this orphan designation evaluation
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: