EU/3/10/832: Orphan designation for the treatment of sickle cell disease



On 23 February 2011, orphan designation (EU/3/10/832) granted by the European Commission to Apotex Europe B.V., the Netherlands, for deferiprone for the treatment of sickle cell disease.

The sponsorship was transferred to Apotex B.V, Netherlands, in May 2019. The sponsor’s address was updated in January 2020.

The sponsorship was transferred to Chiesi Farmaceutici S.p.A., Italy in June 2020.

Key facts

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

Chiesi Farmaceutici S.p.A.
Via Palermo 26/a 
43122 Parma (PR) 

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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