The use of a gene-editing treatment for children from the age of five with severe sickle-cell disease and transfusion-dependent beta thalassaemia (TDT) has been extended by the Medicines and Healthcare Regulatory Agency (MHRA).
The approval allows eligible young patients with the two inherited blood disorders to receive Casegevy (exagamglogene autotemcel), which was previously available for patients over the age of 12.
Severe sickle-cell disease can cause episodes of intense pain, serious infections, and anaemia, while beta-thalassaemia can lead to severe anaemia. Patients with transfusion dependent beta-thalassaemia may require blood transfusions every three to five weeks, alongside lifelong medication and injections.
Casegevy works by editing a faulty gene in a patient’s bone marrow stem cells to enable the body to produce functioning haemoglobin. The patient’s stem cells are collected and modified in a laboratory before being infused back into the body.
Patients undergo conditioning treatment to prepare their bone marrow before the modified cells are returned. They may then need to remain in the hospital while the cells establish themselves and begin producing red blood cells with functioning haemoglobin.
Julian Beach, MHRA's Executive Director of Operations, said: “Severe sickle cell disease and beta–thalassaemia are serious, lifelong conditions that in some cases can be fatal. This approval means that this innovative gene-editing treatment can now be used in eligible children from age of five, extending this treatment option to younger patients after careful assessment of the available evidence on the treatment’s safety, quality and effectiveness.”
Approximately 15,000 people are thought to be living with sickle-cell disorder in the UK. Access to Casegevy will be limited to those meeting specific eligibility criteria.
