EU/3/19/2210: Orphan designation for the treatment of beta-thalassaemia intermedia and major

Autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene

Table of contents

Overview

On 17 October 2019, orphan designation EU/3/19/2210 was granted by the European Commission to Vertex Pharmaceuticals (Ireland) Limited, Ireland, for autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the bcl11a gene (also known as CTX001) for the treatment of beta-thalassaemia intermedia and major.

The sponsor’s address was updated in January 2022.

Key facts

Active substance
Autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
Intented use
Treatment of beta-thalassaemia intermedia and major
Orphan designation status
Positive
EU designation number
EU/3/19/2210
Date of designation
17/10/2019
Sponsor

Vertex Pharmaceuticals (Ireland) Limited
Unit 49
Northwood Court
Block F2 Santry
Dublin 9
Ireland
E-mail: vertexmedicalinfo@vrtx.com

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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:

How useful was this page?

Add your rating