EU/3/19/2230: Orphan designation for the treatment of immune thrombocytopenia

Efgartigimod alfa


On 16 December 2019, orphan designation EU/3/19/2230 was granted by the European Commission to Argenx B.V.B.A., Belgium, for efgartigimod alfa for the treatment of immune thrombocytopenia. 

The sponsor's name and address was updated in September 2021.

Key facts

Active substance
Efgartigimod alfa
Intended use
Treatment of immune thrombocytopenia
Orphan designation status
EU designation number
Date of designation

Industriepark-Zwijnaarde 7
9052 Gent
Tel: +32 497 62 46 13

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:

How useful was this page?

Add your rating