26 February 2025 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment of patients with severe sickle cell disease.

Exagamglogene autotemcel is recommended with managed access as an option for the treatment of patients 12 years of age and older with sickle cell disease who have:

• Recurrent vaso-occlusive crises and a βS/βS, βS/β+ or βS/β0 genotype and
• When haematopoietic stem cell transplantation is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available

Exagamglogene autotemcel is only recommended:

• For those who have had at least 2 vaso-occlusive crises per year during the 2 previous years
• If the conditions in the managed access agreement for exagamglogene autotemcel are followed