EU/3/21/2488: Orphan designation for the treatment of Diamond-Blackfan anaemia

Autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene

Table of contents

Overview

This medicine was designated as an orphan medicine for the treatment of Diamond-Blackfan anaemia in the European Union on 20 August 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
Autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene
Intended use
Treatment of Diamond-Blackfan anaemia
Orphan designation status
Positive
EU designation number
EU/3/21/2488
Date of designation
20/08/2021
Sponsor

Yes Pharmaceutical Development Services GmbH
Bahnstrasse 42-46
61381 Friedrichsdorf
Hassia
Germany
E-mail: jhwang@apriligen.com

 

Update history

DateUpdate
June 2022The sponsorship was transferred to Yes Pharmaceutical Development Services GmbH, Germany in June 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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