Vertex Pharmaceuticals has secured MHRA authorisation and reimbursement for expanded use of Casgevy for eligible children aged 5 to under 12 years with severe sickle cell disease or transfusion‑dependent beta thalassemia.
The decision makes exagamglogene autotemcel the first approved gene therapy available to eligible UK children as young as 5 years for both conditions.
A reimbursement agreement with NHS England, alongside updated guidance from NICE, means eligible patients can now access treatment in England. Vertex said the move strengthens its partnership with the UK health system, which first authorised Casgevy in 2023.
Nicola Massey, Executive Country Manager UK & Ireland, Vertex, said: “Since the UK became the first country to authorize CASGEVY in 2023, our partnership with the UK health system has helped bring this innovation to eligible people living with sickle cell disease and transfusion-dependent beta thalassemia.”
She added: “Today’s news reinforces our long-term commitment to these communities, enabling eligible younger children to access this innovative treatment earlier.”
Professor Josu de la Fuente, Professor of Practice at Imperial College London and Consultant Haematologist at Imperial College Healthcare NHS Trust, explained: “For children living with sickle cell disease or transfusion-dependent beta thalassemia, complications generally begin at a very young age and have lasting consequences.”
He continued: “Today’s approval is an important advance and offers new hope to eligible children and their families. It enables us to consider younger children for treatment earlier, before disease-related complications lead to lasting organ damage.”
Vertex is working with health authorities in Wales, Scotland and Northern Ireland to extend access for eligible patients as quickly as possible.
