exagamglogene autotemcel at a glance
Medicine class
Patient-specific, ex-vivo gene-edited autologous stem cell therapy.
Licensed uses
Selected patients aged 12 years and over with transfusion-dependent beta-thalassaemia or sickle cell disease with recurrent vaso-occlusive crises.
Administration
Intravenous infusion in an authorised specialist treatment centre after collection and preparation of the patient’s stem cells.
Follow-up
Long-term registry follow-up is expected while evidence about long-term safety and effectiveness continues to be collected.
What is exagamglogene autotemcel used for?
UK-licensed uses are:
Transfusion-dependent beta-thalassaemia in patients aged 12 years and over when haematopoietic stem cell transplantation is appropriate and a matched related donor is unavailable.
Sickle cell disease in patients aged 12 years and over with recurrent vaso-occlusive crises and an eligible beta-globin genotype, when haematopoietic stem cell transplantation is appropriate and a matched related donor is unavailable.
How does exagamglogene autotemcel work?
CRISPR/Cas9 editing reduces BCL11A activity in developing red blood cells. This increases fetal haemoglobin, helping red cells work more effectively in beta-thalassaemia and reducing sickling in sickle cell disease.
How to take exagamglogene autotemcel
This treatment is not taken at home. The patient’s stem cells are collected, edited and tested, then returned by intravenous infusion in an authorised treatment centre after bone-marrow conditioning. Specialist hospital monitoring continues while blood-cell production recovers.
Staff verify the patient-specific product before infusion.
All supplied patient-specific vials must be accounted for and handled by trained staff.
Recipients must never donate blood, organs, tissues or cells after treatment.
exagamglogene autotemcel side effects
Common or expected effects
- Low white cells, red cells or platelets
- Fever, headache and tiredness
- Nausea, vomiting, diarrhoea, constipation or reduced appetite
- Mouth or other mucosal inflammation
- Abdominal, bone or muscle pain
- Nosebleeds, skin spots or bruising
- Hair loss and skin changes
- Changes in liver tests or blood electrolytes
Get urgent medical advice
- Severe infection or failure of neutrophil recovery
- Prolonged low platelets with serious bleeding
- Serious allergic reaction during or after infusion
- Severe breathing problems, including acute respiratory distress
- Haemophagocytic lymphohistiocytosis or engraftment syndrome
- Serious complications from conditioning or stem cell transplantation
exagamglogene autotemcel in pregnancy
It must not be administered during pregnancy because of risks from conditioning; pregnancy data are unavailable. Pregnancy testing and effective contraception are required around treatment. Conditioning can cause infertility, so fertility-preservation options should be discussed beforehand.
exagamglogene autotemcel while breastfeeding
Breastfeeding must stop during conditioning. It is unknown whether exagamglogene autotemcel can reach a breastfed child; restarting after infusion requires an individual discussion with the specialist.
exagamglogene autotemcel interactions
The specialist team must review all medicines and vaccines before mobilisation, conditioning and infusion.
Hydroxyurea or hydroxycarbamide
Use around mobilisation and conditioning requires specialist management; experience after infusion is limited.
Voxelotor or crizanlizumab
Use around mobilisation and conditioning requires specialist management because interactions with preparative medicines are unknown.
Iron-chelating medicines
Require specialist management around conditioning and recovery because of interaction and bone-marrow suppression concerns.
Common questions about exagamglogene autotemcel
Answers are fully visible for fast scanning and source review.
What is exagamglogene autotemcel made from?
The patient’s own collected blood-forming stem cells, edited outside the body and cryopreserved before being returned.
Why are stem cells collected before treatment?
Some are used for gene editing and manufacture; untreated cells are stored as rescue cells if the edited product cannot be used or fails to engraft.
Can it be given at home?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.