following a full submission assessed under the orphan medicine process:
exagamglogene autotemcel (Casgevy®) is accepted for use within NHSScotland.
Indication under review: for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom haematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available.
In a single-arm open-label study, 97% (28/29) of patients remained free from severe vaso-occlusive crises for at least 12 consecutive months after receiving an exagamglogene autotemcel (exa-cel) infusion.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Medicine details
- Medicine name:
- exagamglogene autotemcel (Casgevy)
- SMC ID:
- SMC2852
- Indication:
Treatment of sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs) who have βS/βS, βS/β0 or βS/β+, for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.
- Pharmaceutical company
- Vertex Pharmaceuticals Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Full
- Status
- Accepted
- Date advice published
- 19 January 2026