EU/3/18/2034: Orphan designation for the treatment of neurodegeneration with brain iron accumulation



On 27 June 2018, orphan designation (EU/3/18/2034) was granted by the European Commission to Apotex Europe B.V., the Netherlands, for deferiprone for the treatment of neurodegeneration with brain iron accumulation.

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 May 2020.

Key facts

Active substance
Intended use
Treatment of neurodegeneration with brain iron accumulation
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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