EU/3/21/2505: Orphan designation for the treatment of mucopolysaccharidosis type I

Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase

Table of contents

Overview

This medicine was designated as an orphan medicine for the treatment of mucopolysaccharidosis type I in the European Union on 15 October 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
Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase
Intended use
Treatment of mucopolysaccharidosis type I
Orphan designation status
Positive
EU designation number
EU/3/21/2505
Date of designation
15/10/2021
Sponsor

TMC Pharma (EU) Limited
7A Durands Court
45 Parnell Street
Waterford
Co. Waterford
X91 P381
Ireland
Tel. + 353766705745
E-mail: info@tmcpharma.com

Update history

DateUpdate
May 2022The sponsor’s address was updated in May 2022.

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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