Autologous CD34+ cells encoding βA-T87Q-globin gene

This medicine is authorised for use in the European Union.


Zynteglo is a medicine used to treat a blood disorder known as beta thalassaemia in patients 12 years and older who require regular blood transfusions.

People with this genetic condition cannot make enough beta-globin, a component of haemoglobin, the protein in red blood cells that carries oxygen around the body. As a result, these patients have low red blood cell levels and need frequent blood transfusions.

Zynteglo is used in patients who do not completely lack beta-globin and who are eligible for stem cell transplantation but do not have a matching related donor.

Beta thalassaemia is rare, and Zynteglo was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 24 January 2013.

Zynteglo contains as its active substance stem cells taken from the patients that have been genetically modified to contain a working gene for beta-globin.

This EPAR was last updated on 03/06/2019

Authorisation details

Product details
Agency product number
Active substance
Autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin BB305 lentiviral vector encoding the beta-A-T87Q-globin gene
International non-proprietary name (INN) or common name
Autologous CD34+ cells encoding βA-T87Q-globin gene
Therapeutic area (MeSH)
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
bluebird bio (Netherlands) B.V.
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Stadsplateau 7
3521 AZ Utrecht
The Netherlands

Product information

Zynteglo - EMEA/H/C/003691 -


Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.

Pharmacotherapeutic group

Other hematological agents

Therapeutic indication

Zynteglo is indicated for the treatment of patients 12 years and older with transfusion-dependent β thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

Assessment history

How useful was this page?

Add your rating
1 rating