EU/3/21/2414: Orphan designation for the treatment of mucopolysaccharidosis type I

Alpha-L-iduronidase fused to Fab fragment of a humanised monoclonal antibody targeting human transferrin receptor

Table of contents

Overview

This medicine was designated as an orphan medicine for the treatment of mucopolysaccharidosis type I in the European Union on 26 March 2021.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

Key facts

Active substance
Alpha-L-iduronidase fused to Fab fragment of a humanised monoclonal antibody targeting human transferrin receptor
Intended use
Treatment of mucopolysaccharidosis type I
Orphan designation status
Positive
EU designation number
EU/3/21/2414
Date of designation
26/03/2021
Sponsor

Artemida Pharma Europe Limited
The Black Church
Saint Mary's Place North
Dublin 7 D07 P4AX
Ireland
Tel. +353 1437 2313
E-mail: artemida@artemidapharma.com

 

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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