MHRA 批准 Casgevy 用于 5 岁及以上严重遗传性血液病患儿

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved an extension to the use of Casgevy (exagamglogene autotemcel), allowing children aged five and over with severe sickle-cell disease and transfusion-dependent β-thalassemia to receive the gene editing treatment. 

The treatment was previously authorised in the UK for patients 12 and over with these serious inherited blood disorders. The latest approval extends its use to younger children who meet the eligibility criteria. 

Both severe sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.  

In people with severe sickle cell disease, this genetic error can lead to attacks of very severe pain, serious and life-threatening infections, and anaemia (whereby your body has difficulty carrying oxygen).  

In people with β-thalassaemia, it can lead to severe anaemia. Patients often need a blood transfusion every 3 to 5 weeks, and injections and medicines throughout their lives.  

Casgevy works by editing a faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. The stem cells are collected from the patient, edited in a laboratory and then infused back into the body following conditioning treatment.  

Julian Beach, MHRA Executive Director Operations, said:    

“Severe sickle cell disease and β-thalassemia are serious, lifelong conditions that in some cases can be fatal.  

“This approval means this innovative gene-editing treatment can now be used in eligible children from the age of five, extending this treatment option to younger patients. It is based on careful assessment of the available evidence on the treatment’s safety, quality and effectiveness. 

“As with all medicines, we will continue to closely monitor the safety and effectiveness of Casgevy.”  

Side effects associated with this treatment include nausea, fatigue, fever and increased risk of infection. 

Before treatment with Casgevy, patients undergo conditioning treatment to prepare the bone marrow for the modified stem cells. Following the infusion, patients may need to remain in hospital while the treated cells establish themselves in the bone marrow and begin producing red blood cells with functioning haemoglobin. 

Notes to Editors   

• The marketing authorisation extension was granted on 29 October 2026 to Vertex Pharmaceuticals (Europe) Ltd.   

• Approximately 15,000 people in the UK have sickle cell disorder. 

• More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.   

• The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.   

• The MHRA is an executive agency of the Department of Health and Social Care.   

• For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.