EU/3/20/2341: Orphan designation for the treatment of Fabry disease

Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha

Overview

On 19 October 2020, orphan designation EU/3/20/2341 was granted by the European Commission to Clinical Technology Centre (Ireland) Limited, Ireland, for autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha (also known as AVR-RD-01) for the treatment of Fabry disease.

Key facts

Active substance
Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha
Intended use
Treatment of Fabry disease
Orphan designation status
Positive
EU designation number
EU/3/20/2341
Date of designation
19/10/2020
Sponsor

PPD Bulgaria EOOD
Tsarigradsko Shose Boulevard 115
Sofia 1784
Bulgaria
E-mail: carmen.vieira@avrobio.com

Review of designation

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

Update history

DateUpdate
June 2022The sponsorship was transferred to PPD Bulgaria EOOD, Bulgaria 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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