EU/3/20/2278: Orphan designation for the treatment of immune thrombocytopenia
Table of contents
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.
Treatment of immune thrombocytopenia
|Orphan designation status||
|EU designation number||
|Date of designation||
Genzyme Europe B.V.
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
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: