Zynteglo

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Autologous CD34+ cells encoding βA-T87Q-globin gene

Authorised
This medicine is authorised for use in the European Union.

Overview

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 14/11/2019

Authorisation details

Product details
Name
Zynteglo
Agency product number
EMEA/H/C/003691
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)
beta-Thalassemia
Anatomical therapeutic chemical (ATC) code
B06A
Accelerated assessmentAccelerated assessment

This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment.

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.

Conditional approvalConditional approval

This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.

OrphanOrphan

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.
Revision
1
Date of issue of marketing authorisation valid throughout the European Union
29/05/2019
Contact address

Stadsplateau 7
3521 AZ Utrecht
The Netherlands

Product information

31/10/2019 Zynteglo - EMEA/H/C/003691 - IB/0003

Contents

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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

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