Zynteglo
betibeglogene autotemcel
Table of contents
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.
-
List item
Zynteglo : EPAR - Medicine overview (PDF/91.13 KB)
First published: 03/06/2019
EMA/205979/2019 -
-
List item
Zynteglo : EPAR - Risk-management-plan summary (PDF/220.91 KB)
First published: 03/06/2019
Last updated: 04/10/2021
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 |
betibeglogene autotemcel
|
| Therapeutic area (MeSH) |
beta-Thalassemia
|
| Anatomical therapeutic chemical (ATC) code |
B06A
|
Accelerated 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 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 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. |
Orphan |
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 |
5
|
| Date of issue of marketing authorisation valid throughout the European Union |
29/05/2019
|
| Contact address |
Stadsplateau 7 |
Product information
09/12/2021 Zynteglo - EMEA/H/C/003691 - N/0030
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
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.