Zynteglo
- Procedure started
- Under evaluation
- PRAC recommendation
- CAT draft opinion
- CHMP opinion
- European Commission final decision
Overview
CHMP endorses review finding no link between viral vector in Zynteglo and blood cancer
On 22 July 2021, EMA’s human medicines committee (CHMP) endorsed findings of a review which concluded that there is no evidence Zynteglo causes a blood cancer known as acute myeloid leukaemia (AML).
Zynteglo, a gene therapy for the blood disorder beta thalassaemia, uses a viral vector (or modified virus) to deliver a working gene into the patient’s blood cells.
The review considered two cases of AML in patients treated with an investigational medicine, bb1111, in a clinical trial for sickle cell disease. Although there have been no reports of AML with Zynteglo, both medicines use the same viral vector and there was a concern that the vector may be implicated in the development of the cancer (insertional oncogenesis).
The review by EMA’s safety committee (PRAC) supported by experts from the Committee for Advanced Therapies (CAT) found that the viral vector was unlikely to be the cause. In one of the patients, the viral vector was not present in the cancer cells, and in the other patient it was present at a site (VAMP4) that does not appear to be involved in cancer development.
After examining all the evidence, it was clear that more plausible explanations for the AML cases included the conditioning treatment the patients received to clear out bone marrow cells and the higher risk of blood cancer in people with sickle cell disease.
Patients having Zynteglo treatment for beta thalassaemia also need conditioning treatment to clear out their bone marrow cells before receiving Zynteglo. Healthcare professionals should therefore explicitly inform patients receiving Zynteglo of the increased risk of blood cancers from medicines used in conditioning treatments.
The CHMP agreed on an update of recommendations for monitoring patients. Healthcare professionals should now check their patients for signs of blood cancers at least once a year for 15 years.1
The CHMP concluded that the benefits of Zynteglo continue to outweigh its risks. As for all medicines, the EMA will monitor any new data on its safety and update advice for patients and healthcare professionals when necessary.
1 The previous recommendation was for healthcare professionals to check for signs of cancer once a year.
Key facts
About this medicine
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Approved name |
Zynteglo
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International non-proprietary name (INN) or common name |
betibeglogene autotemcel
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Associated names |
Zynteglo
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Class |
Haematological agents
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About this procedure
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Current status |
European Commission final decision
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Reference number |
EMEA/H/A-20/1504/C/003691/0023
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Type |
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. |
Authorisation model |
Centrally authorised product(s)
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Decision making model |
PRAC-CAT-CHMP-EC
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Key dates and outcomes
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Procedure start date |
11/03/2021
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PRAC recommendation date |
09/07/2021
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CHMP opinion/CMDh position date |
22/07/2021
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EC decision date |
16/09/2021
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Outcome |
Risk minimisation measures
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All documents
Procedure started
Zynteglo Article-20 referral - PRAC List of questions (PDF/796.86 KB)
First published: 12/03/2021
EMA/PRAC/104560/2021
Zynteglo Article-20 referral - Notification (PDF/278.35 KB)
First published: 12/03/2021
Zynteglo Article-20 referral - Timetable for the procedure (PDF/683.15 KB)
First published: 12/03/2021
Last updated: 11/06/2021
EMA/PRAC/104559/2021 Rev. 1
Zynteglo Article-20 referral - Review of thalassaemia medicine Zynteglo started (PDF/701.84 KB)
First published: 12/03/2021
EMA/127031/2021
Recommendation provided by Pharmacovigilance Risk Assessment Committee
Opinion provided by Committee for Medicinal Products for Human Use
European Commission final decision
Zynteglo Article-20 referral - PRAC assessment report (PDF/1.19 MB)
First published: 04/10/2021
EMA/418200/2021
Zynteglo Article-20 referral - Annex III (PDF/1.56 MB)
First published: 04/10/2021
Zynteglo Article-20 referral - Annex IV (PDF/707.77 KB)
First published: 04/10/2021
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.
News
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23/07/2021
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23/07/2021
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09/07/2021
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09/07/2021
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11/06/2021
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07/05/2021
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09/04/2021