Exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals) is indicated for 'the treatment of transfusion-dependent β‑thalassemia in patients 12 years of age and older 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 for exagamglogene autotemcel is £1,651,000 per course of treatment.
The company has a commercial arrangement. This makes exa‑cel available to the NHS with a discount. The size of the discount is commercial in confidence.