NICE Recommends Vertex's £1.6 Million One-Time Gene Therapy Casgevy for Beta-Thalassaemia | iPharmaCenter

Patients in England suffering from severe beta-thalassaemia will soon have access to a groundbreaking one-time gene therapy, exagamglogene autotemcel, making them among the first in Europe to benefit from this treatment.

The final draft guidance from NICE endorses exagamglogene autotemcel, also known as "exa-cel" and branded as Casgevy by Vertex, for patients aged 12 and older who have severe beta-thalassaemia and require regular blood transfusions. This recommendation applies specifically when a blood and bone marrow transplant is deemed appropriate, but a suitable donor is unavailable.

NICE Recommends Vertex's £1.6 Million One-Time Gene Therapy Casgevy for Beta-Thalassaemia

Exa-cel is the world’s first gene therapy utilizing CRISPR technology, marking a significant advancement as the first gene therapy in Europe aimed at treating severe beta-thalassaemia.

Patients with the most severe form of beta-thalassaemia require regular blood transfusions to manage their condition. This severe type can lead to delayed growth, bone abnormalities, endocrine issues, and significantly impact both the quality and duration of life.

The independent committee received insights from patient experts who described the substantial impact that frequent blood transfusions and their associated side effects and complications have on their work, family life, and social relationships.

Exa-cel, priced at £1,651,000 per treatment course, operates by extracting and modifying (editing) the faulty gene in the patient’s bone marrow stem cells, enabling the body to produce functioning hemoglobin.

These edited cells are then reintroduced into the patient, with the advantage that, because the cells are their own, there is no risk of rejection by the body.

Exa-cel will be made available through the Innovative Medicines Fund, allowing for the collection of more data on its clinical and cost-effectiveness. This arrangement ensures that up to 460 eligible patients can access the treatment immediately, speeding up its availability.

Beta-thalassaemia is a hereditary blood disorder caused by a genetic mutation that impairs or halts the production of healthy red blood cells and hemoglobin, the protein responsible for transporting oxygen throughout the body.

In the UK, beta-thalassaemia primarily affects individuals of Pakistani, Indian, and Bangladeshi descent.