On 19 November 2018, orphan designation (EU/3/18/2093) was granted by the European Commission to Sanquin Plasma Products B.V., the Netherlands, for human apotransferrin for the treatment of beta-thalassaemia intermedia and major.

Key facts

Active substance
human apotransferrin
Disease / condition
Treatment of beta thalassaemia intermedia and major
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Sanquin Plasma Products B.V.
Plesmanlaan 125
1066 CX Amsterdam
The Netherlands
Tel. +31 20 512 30 00

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;

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