EU/3/20/2356: Orphan designation for the treatment of sickle cell disease

Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene

Table of contents

Overview

On 13 November 2020, orphan designation EU/3/20/2356 was granted by the European Commission to Clinical Technology Centre (Ireland) Limited, Ireland, for autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene (also known as ARU-1801) for the treatment of sickle cell disease.

Please note that this product was withdrawn from the Union Register of orphan medicinal products in September 2022 on request of the Sponsor.

Key facts

Active substance
Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene
Intended use
Treatment of sickle cell disease
Orphan designation status
Withdrawn
EU designation number
EU/3/20/2356
Date of designation
13/11/2020
Sponsor

PPD Bulgaria EOOD
Tsarigradsko Shose Boulevard 115
Sofia 1784
Bulgaria
E-mail: moya.daniels@aruvant.com

Update history

DateUpdate
September 2022Product withdrawn from the Union Register of orphan medicinal products on request of the Sponsor.
April 2022The sponsorship was transferred to PPD Bulgaria EOOD, Bulgaria, in April 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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