This medicine is now known as tralesinidase alfa.
On 15 January 2015, orphan designation (EU/3/14/1422) was granted by the European Commission to BioMarin Europe Limited, United Kingdom, for chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 for the treatment of mucopolysaccharidosis type IIIB.
chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa)
Treatment of mucopolysaccharidosis type IIIB
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|January 2022||The sponsorship was transferred to Allievex Europe B.V., Netherlands.|
|April 2020||The sponsorship was transferred to IQVIA RDS Spain S.L., Spain.|
|January 2019||The sponsorship was transferred to BioMarin International Limited, Ireland.|
Documents related to this orphan designation evaluation
EU/3/14/1422: Public summary of opinion on orphan designation: Chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 for the treatment of mucopolysaccharidosis ... (PDF/131.6 KB)
First published: 06/03/2015
Last updated: 06/03/2015
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: