Indication
For the treatment of transfusion-dependent β-thalassaemia in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate and a human leukocyte antigen (HLA)-matched related haematopoietic stem cell (HSC) donor is not available.
Medicine details
- Medicine name:
- exagamglogene autotemcel (exa-cel) (Casgevy)
- SMC ID:
- SMC2709
- Pharmaceutical company
- Vertex Pharmaceuticals Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Ultra-orphan initial assessment
- Publication due date:
- TBC
- SMC meeting date:
- TBC
- Patient group submission deadline:
- TBC