posted 8th August 2024
On this day (08.08.2024) the National Institute for Health and Care Excellence (NICE) approves NHS use of Casgevy ™ [Exagamglogene Autotemcel ('Exa-Cel')] for the treatment of Transfusion-dependent beta Thalassaemia (TDT) in patients of 12 years and older.
NHS England will be among the first of health systems in the world to offer this breakthrough treatment which has been developed by Vertex Pharmaceuticals. This therapy uses the innovative CRISPR/Cas9 gene editing tool which won the inventors the Nobel Prize in 2020. The one-time treatment offers much hope to patients, due to its potential to offer a disease-free status for people with the life-limiting genetic condition of TDT. It is anticipated that patients with Sickle Cell Disorder, another life-limiting genetic haemoglobin disorder (with a substantially large population in the UK), will also have access to this therapy in the near future.
The NHP, along with many other haemoglobinopathy groups, teams, patients and allies, are amongst the stakeholders who have been advocating for and liaising with NICE towards their of approval for the Exa-Cel treatments.
For further information, please see the following NHS England link: https://www.england.nhs.uk/2024/08/gene-editing-therapy-that-could-cure-blood-disorder-thalassaemia-for-nhs-patients/
