Hundreds of Patients in England To Benefit From the World's First Gene Editing Therapy

NICE’s final draft guidance recommends exagamglogene autotemcel (“exa-cel”, also called Casgevy and made by Vertex) for people 12 years and over with severe beta-thalassaemia who need regular blood transfusions to manage their condition and when a blood and bone marrow transplant is suitable but no donor is available. 

Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia.   

Beta-thalassaemia is an inherited blood disorder caused by a genetic mutation that reduces or prevents production of healthy red blood cells and haemoglobin (the protein found in red blood cells that carries oxygen around the body). 

In the UK the condition mainly affects people of Pakistani, Indian and Bangladeshi ethnic origin.

People with the most severe type of beta thalassaemia need regular blood transfusions. Severe beta-thalassaemia can cause delayed growth, bone problems, problems with endocrine development and affect quality and length of life. 

The independent committee heard from patient experts about the significant effect on work, family and friends that intense blood transfusions and their associated side effects and complications can have.  

"Although there are some uncertainties in the evidence for its long-term benefits, the committee felt exa-cel could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions," said Helen Knight, director of medicines evaluation at NICE.

Costing £1,651,000 per course of treatment at its list price, exa-cel works by first removing and then modifying (editing) the faulty gene in a patient’s bone marrow stem cells so the body produces functioning haemoglobin – the protein in red blood cells that carries oxygen around the body. The edited cells are then put back into the patient. As exa-cel involves people receiving their own edited cells, there is no risk of their body rejecting them. 

"Today’s final draft guidance also addresses our aim of reducing health inequalities associated with conditions like transfusion-dependent beta-thalassaemia. In doing so it demonstrates our ability to address these issues proportionately and flexibly as part of our standard process, ensuring access to innovative, effective treatments that provide value for money for taxpayers and the NHS," said Helen Knight, director of medicines evaluation at NICE.

Exa-cel will be available through the Innovative Medicines Fund so that more data about its clinical and cost-effectiveness can be collected. This means it will be funded immediately to accelerate rollout for up to 460 people eligible for the treatment. 

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