• Procedure started
  • Under evaluation
  • PRAC recommendation
  • CHMP opinion
  • European Commission final decision
Current status:
Procedure started


Review of thalassaemia medicine Zynteglo started

EMA has begun a safety review of the medicine Zynteglo, a gene therapy authorised to treat the rare blood condition beta thalassaemia.

The review follows a case of acute myeloid leukaemia, a cancer of the blood, in a patient treated with a related investigational medicine, bb1111. This medicine uses the same modified virus (known as a viral vector) as Zynteglo, to deliver a gene into body cells. However, bb1111 is under development to treat sickle cell disease rather than thalassaemia. As well as the leukaemia case, 2 other patients given bb1111 developed another blood disorder, myelodysplastic syndrome, which progressed to leukaemia in one of them.

So far no cases of leukaemia have been reported with Zynteglo itself. However, the possibility that this type of treatment could cause a blood cancer (insertional oncogenesis) had been recognised as a potential risk when it was authorised, and patients who receive the medicine are followed up long-term and monitored in a registry. Given this, and since bb1111 works in the same way, the company responsible for developing both medicines has paused supply of Zynteglo while the evidence is examined to understand whether the cancer might be related to their treatment. No other authorised medicines use the same viral vector, which is based on a type of virus called a lentivirus.

EMA’s safety committee, PRAC, will now examine the evidence thoroughly at EU level, working closely with experts from EMA’s Committee for Advanced Therapies (CAT), which is responsible for assessing this type of medicine, and decide on any relevant regulatory action for Zynteglo.

Zynteglo is currently only available from 2 centres in the EU, and only one patient has received the medicine outside the context of clinical studies. If patients treated with Zynteglo or bb1111 have any concerns they should contact the doctor supervising their treatment.

Key facts

About this medicine
Approved name
International non-proprietary name (INN) or common name
betibeglogene autotemcel
Associated names
Haematological agents
About this procedure
Current status
Procedure started
Reference number
Article 20 procedures

This type of procedure is triggered for medicines that have been authorised via the centralised procedure in case of quality, safety or efficacy issues.

Decision making model
Authorisation model
Centrally authorised product(s)
Key dates and outcomes
Procedure start date

All documents

Procedure started

Description of documents published

Please note that some of the listed documents apply only to certain procedures.

  • Overview - lay-language summary of the stage of the procedure
  • Notification – a letter from a Member State, the European Commission or the marketing authorisation holder requesting the initiation of the procedure
  • Scientific background – further background information from the triggering Member State on the issues leading to the initiation of the procedure (if applicable)
  • List of questions – questions agreed by the Committee requesting further information from the marketing authorisation holder(s) / applicant(s) to evaluate the issues identified
  • Timetable for the procedure – agreed timeframe to respond to the list of questions, to assess the issues and to adopt a conclusion
  • List of medicines concerned by the procedure – medicine(s) / active substance(s) concerned, and marketing authorisation holder(s) / applicant(s)
  • List of questions to be addressed by the stakeholders – call for data to be submitted by stakeholders (e.g. healthcare professionals, patient organisations, individual patients) (if applicable)
  • Stakeholder submission form – form to be used by stakeholders to submit data (if applicable)
  • Scientific conclusions – scientific conclusions of the PRAC and/or CHMP and/or CMDh
  • Assessment report – PRAC or CHMP assessment and conclusions on the issues investigated, including divergent positions (if applicable)
  • Divergent positions – divergent positions of the CHMP or CMDh members for pharmacovigilance procedures (if applicable)
  • Changes to the summary of product characteristics, labelling and package leaflet (amended sections or fully revised version) (if applicable)
  • Condition(s) to the marketing authorisation(s) – condition(s) for the safe and effective use of the medicine(s) (if applicable)
  • Condition for lifting the suspension – condition to be fulfilled for the suspension of the marketing authorisation(s) to be lifted (if applicable)
  • Timetable for implementation of CMDh position – agreed timeframe to submit and finalise the variation(s) implementing the outcome of the procedure (if applicable)

Note that older documents may have different titles.


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