Exagamglogene autotemcel for treating severe sickle cell disease in people 12 years and over

Exagamglogene autotemcel (Casgevy, Vertex) is indicated for 'the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the β^S/β^S, β^S/β⁺ or β^S/β⁰ genotype, for whom haematopoietic stem cell transplantation is appropriate and a human leukocyte antigen-matched related haematopoietic stem cell donor is not available'.

The dosage schedule is available in the summary of product characteristics for exagamglogene autotemcel.

The list price per treatment for a single dose of exagamglogene autotemcel is £1,651,000.

The company has a commercial arrangement. This makes exagamglogene autotemcel available to the NHS with a discount. The size of the discount is commercial in confidence.