EU/3/14/1422

About

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.

The sponsorship was transferred to BioMarin International Limited, Ireland, in January 2019 and subsequently to IQVIA RDS Spain S.L., Spain, in April 2020.

Key facts

Active substance
chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa)
Disease / condition
Treatment of mucopolysaccharidosis type IIIB
Date of first decision
15/01/2015
Outcome
Positive
EU designation number
EU/3/14/1422

Review of designation

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

Sponsor's contact details

IQVIA RDS Spain S.L.
Calle De Juan Esplandiu 11 Planta 6
28007 Madrid
Spain
Tel. +34 627057039
E-mail: inquiries@allievex.com

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.

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