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.
Treatment of neurodegeneration with brain iron accumulation
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Review of designation
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: