11 September 2024 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment of patients 12 years of age and older with transfusion-dependent beta thalassaemia.

Exagamglogene autotemcel is recommended with managed access as an option for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassaemia when haematopoietic stem cell transplantation is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available and only if the conditions in the managed access agreement for exagamglogene autotemcel are followed.

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