Current status: Answered by Jenni Minto on 11 March 2025
To ask the Scottish Government, following the treatment, exagamglogene autotemcel, which is a gene-edited therapy manufactured in Scotland, being approved for use in England to treat (a) transfusion dependent thalassaemia and (b) sickle cell disease, what engagement it is having with the NHS and suppliers toward making this available in Scotland, also broken down by what date it will be made available to people eligible for it, if approved.
The Scottish Medicines Consortium (SMC) has received a submission through the Ultra-Orphan Pathway from the marketing authorisation holder, Vertex Pharmaceuticals, for exagamglogene autotemcel (Casgevy®) for the treatment of transfusion-dependent ß-thalassaemia in patients 12 years of age and older. A decision on whether Casgevy® is accepted on to the Ultra-Orphan Pathway for this indication will be published on the SMC website in due course.
The SMC has not yet received a submission from Vertex Pharmaceuticals for Casgevy® for the treatment of sickle cell disease (SCD). I refer the member to the answer to question S6W-34796 on 25 February 2025. All answers to written Parliamentary Questions are available on the Parliament's website, the search facility for which can be found at https://www.parliament.scot/chamber-and-committees/written-questions-and-answers.