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.
recombinant human interleukin-3 truncated diphtheria toxin fusion protein
Treatment of blastic plasmacytoid dendritic cell neoplasm
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Review of designation
Documents related to this orphan designation evaluation
EU/3/15/1567: Public summary of opinion on orphan designation: Recombinant human interleukin-3 truncated diphtheria toxin fusion protein for treatment of blastic plasmacytoid dendritic cell neoplasm (PDF/126.65 KB)
First published: 05/01/2016
Last updated: 05/01/2016
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: