EU/3/15/1567: Orphan designation for the treatment of blastic plasmacytoid dendritic cell neoplasm
recombinant human interleukin-3 truncated diphtheria toxin fusion protein
Table of contents
Overview
On 11 November 2015, orphan designation (EU/3/15/1567) was granted by the European Commission to Spector Consulting SAS, France, for recombinant human interleukin-3 truncated diphtheria toxin fusion protein (also known as SL-401) for the treatment of blastic plasmacytoid dendritic cell neoplasm.
The sponsorship was transferred to TMC Pharma (EU) Limited, Ireland, in November 2018 and to Stemline Therapeutics B.V. in November 2019.
Recombinant human interleukin-3 truncated diphtheria toxin fusion protein has been authorised in the EU as Elzonris since 7 January 2021.
Key facts
Active substance |
recombinant human interleukin-3 truncated diphtheria toxin fusion protein
|
Intended use |
Treatment of blastic plasmacytoid dendritic cell neoplasm
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/15/1567
|
Date of designation |
11/11/2015
|
Sponsor |
Stemline Therapeutics B.V. |
Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of product at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the
orphan medicine assessment report
.
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: