Beta-Thalassemia Gene Therapy Approved for NHS England

The United Kingdom’s National Institute for Health and Care Excellence approved the first gene therapy to treat transfusion-dependent beta-thalassemia (TDT) for use on England’s National Health Service (NHS) beginning Aug. 8.

The therapy, exagamglogene autotemcel (Casgevy, Vertex), is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy. It involves editing a patient’s own hematopoietic stem and progenitor cells at the erythroid-specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin in red blood cells, significantly reducing or eliminating the need for transfusions in patients with TDT.

NHS England estimates that 460 patients in the country (aged 12 and older) are currently living with TDT and may be eligible for the therapy. Exagamglogene autotemcel costs 1.65 million British pounds per course of treatment and will be funded immediately through the Innovative Medicines Fund to collect more data on its clinical and cost-effectiveness. 

Exagamglogene autotemcel is also approved to treat TDT and sickle cell disease in the United States, European Union and Saudi Arabia.