EU/3/19/2210

Table of contents

About

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.

Key facts

Active substance
Autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
Disease / condition
Treatment of beta-thalassaemia intermedia and major
Date of first decision
17/10/2019
Outcome
Positive
EU designation number
EU/3/19/2210

Sponsor's contact details

Vertex Pharmaceuticals (Ireland) Limited
28-32 Pembroke Street Upper
Dublin 2, D02 EK84
Co. Dublin
Ireland
Tel. +353 1761 7299
E-mail: vertexmedicalinfo@vrtx.com

Patients' organisations

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

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