EU/3/20/2278: Orphan designation for the treatment of immune thrombocytopenia



On 4 June 2020, orphan designation EU/3/20/2278 was granted by the European Commission to Clinical Network Services (NL) B.V., Netherlands, for rilzabrutinib (also known as PRN1008) for the treatment of immune thrombocytopenia.

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

The sponsorship was transferred to Genzyme Europe B.V., Netherlands, in February 2022.

Key facts

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

Genzyme Europe B.V.
Paasheuvelweg 25
1105 BP Amsterdam
The Nederlands
Tel. +31 2024 54000

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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