Zynteglo

Withdrawn

This medicine's authorisation has been withdrawn

betibeglogene autotemcel

Overview

The marketing authorisation for Zynteglo has been withdrawn at the request of the marketing-authorisation holder.

 

Zynteglo : EPAR - Medicine overview

Reference Number: EMA/205979/2019

Zynteglo : EPAR - Risk-management-plan summary

Product information

Zynteglo : EPAR - Product information

Latest procedure affecting product information: N/0030

09/12/2021

This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.

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).

Zynteglo : EPAR - All authorised presentations

Product details

• Name of medicine:
  Zynteglo
• Active substance:
  Autologous CD34+ cell enriched population that contains haematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene
• International non-proprietary name (INN) or common name:
  betibeglogene autotemcel
• Therapeutic area (MeSH):
  beta-Thalassemia
• Anatomical therapeutic chemical (ATC) code:
  B06A

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.

 

Authorisation details

• EMA product number:
  EMEA/H/C/003691
• 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.

• Advanced therapy:

  This medicine is classified as an advanced therapy medicinal product (ATMP): a medicine for human use that is based on genes, tissues or cells. It offers groundbreaking new opportunities for the treatment of disease and injury. For more information, see Advanced therapy medicinal products: Overview.

• 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.

• Marketing authorisation holder:
  bluebird bio (Netherlands) B.V.

  Stadsplateau 7; 3521 AZ Utrecht; The Netherlands

• Opinion adopted:
  28/03/2019
• Marketing authorisation issued:
  29/05/2019
• Revision:
  5

Assessment history

Changes since initial authorisation of medicine

Zynteglo : EPAR - Procedural steps taken and scientific information after authorisation

Zynteglo : EPAR - Scientific conclusions

Zynteglo-H-20-1504-C-003691-0023 : EPAR - PRAC assessment report

AdoptedReference Number: EMA/418200/2021

Initial marketing authorisation documents

Zynteglo : EPAR - Public assessment report

AdoptedReference Number: EMA/56140/2020

Zynteglo : Orphan maintenance assessment report (initial authorisation)

AdoptedReference Number: EMA/263767/2019

CHMP summary of positive opinion for Zynteglo

AdoptedReference Number: EMA/CHMP/166977/2019