EU/3/17/1951: Orphan designation for the treatment of pemphigus

Agammaglobulinaemia tyrosine kinase


On 12 December 2017, orphan designation (EU/3/17/1951) was granted by the European Commission to Clinical Network Services (UK) Ltd, United Kingdom, for agammaglobulinaemia tyrosine kinase (also known as PRN1008) for the treatment of pemphigus.

The sponsorship was transferred to Clinical Network Services (NL) B.V., The Netherlands, in January 2019.

In August 2020, Clinical Network Services (NL) B.V.changed name to Scendea (NL) B.V.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2021 on request of the Sponsor.

Key facts

Active substance
Agammaglobulinaemia tyrosine kinase
Intended use
Treatment of pemphigus
Orphan designation status
EU designation number
Date of designation

Scendea (NL) B.V.
De Cuserstraat 93
1081 CN Amsterdam

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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