EU/3/14/1422: Orphan designation 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)

Overview

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.

Key facts

Active substance
chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa)
Intended use
Treatment of mucopolysaccharidosis type IIIB
Orphan designation status
Positive
EU designation number
EU/3/14/1422
Date of designation
15/01/2015
Sponsor
Allievex Europe B.V.
Floor 4th
Strawinskylaan 4117
Amsterdam
Noord-Holland
1077 ZX
Netherlands
Email: bernie@allievex.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Update history

DateUpdate
January 2022The sponsorship was transferred to Allievex Europe B.V., Netherlands.
April 2020The sponsorship was transferred to IQVIA RDS Spain S.L., Spain.
January 2019The sponsorship was transferred to BioMarin International Limited, Ireland.

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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