Patients in England with severe beta-thalassaemia are to get access to the world’s first CRISPR-based gene therapy, the NHS has announced.
NHS England says this is the first approval of this use of Casgevy – also known as exagamglogene autotemcel, or exa-cel – in Europe. It is to be offered to patients over the age of 12 at seven specialist NHS centres following approval from the National Institute for Health and Care Excellence (NICE).
The treatment can be used when a patient would benefit from a haematopoietic stem cell transplant but there is no suitable donor. The treatment costs £1.6 million a course but the NHS is receiving an unspecified discount.
Helen Knight, director of medicines evaluation at NICE, said: “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.
“Today’s final draft guidance also addresses our aim of reducing health inequalities associated with conditions like transfusion-dependent beta-thalassaemia.”
Professor Bola Owolabi, director of the National Healthcare Inequalities Improvement Programme at NHS England, said: “This is an incredibly exciting step forward in the treatment of thalassaemia and could drastically change the lives of those living with what can be an extremely painful condition.
“We hope this ground-breaking treatment enables people with beta thalassaemia to live longer, more independent and higher quality lives – without the need for regular treatments and hospital appointments and without pain, fatigue and other side effects that can come with this severe and life-limiting condition.”
Romaine Maharaj, executive director of The UK Thalassaemia Society, said: “With NICE’s approval of gene therapy for transfusion dependent thalassaemia under the NHS managed access scheme, we stand on the brink of a revolutionary breakthrough. This transformative treatment offers patients a life-changing opportunity, enabling them to repair their own cells and embrace a future free from the challenges of their condition.”
Patient Kirthana Balachandran, aged 21, a medical student from West London, said: “Gene therapy would mean a lot to me – it would be a life-changing treatment. I’m only 21 and the idea of depending on transfusions for quite literally the rest of my life is daunting.”
Source: NHS England/NICE
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