Ultra Orphan Assessment
The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra-orphan framework:
Indication under review: for the treatment of transfusion-dependent beta-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.
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Scottish Government Medicines Policy Branch will notify Health Boards when this medicine is available for prescribing within the ultra-orphan pathway. Meantime any requests to access treatment should be considered through local non-formulary processes.
Medicine details
- Medicine name:
- exagamglogene autotemcel (exa-cel) (Casgevy)
- SMC ID:
- SMC2709
- 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.
- Pharmaceutical company
- Vertex Pharmaceuticals Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Ultra-orphan initial assessment
- Date Published
- 12 May 2025