People living with a rare genetic blood disorder in England will be offered a “life-changing” new treatment on the NHS, in what has been described as a historic moment.
Casgevy, which is a one-off gene therapy, has been approved for use on the NHS in England for people living with a severe form of thalassaemia.
The treatment was approved by the National Institute for Health and Care Excellence (Nice) on Wednesday, meaning that about 460 patients currently living with transfusion-dependent beta thalassaemia could be eligible for the treatment.
Thalassaemia is the name for a group of inherited blood disorders in which too little haemoglobin is produced by the body. It affects about 2,300 people in the UK, who are mainly from a Mediterranean, Asian, or Middle Eastern background. Many people with beta thalassaemia are not expected to live beyond their 50s.
Casgevy works by editing a gene in a recipient’s bone marrow stem cells so that the body produces functioning haemoglobin.
The therapy is hoped to be a lifetime cure. In international clinical trials, 93% of patients with beta thalassaemia did not need a blood transfusion for at least a year after having the treatment.
Amanda Pritchard, the chief executive of the NHS, said: “This is a historic moment for people living with beta thalassaemia, with a potential cure for those facing this debilitating disorder now available on the NHS.
“Ordinarily, patients experience painful side effects and undergo regular transfusions, which severely impact their quality of life, but this therapy offers people a life free from that, as well as the hope of living longer, which is truly amazing news.
“This is the latest in a series of revolutionary gene therapies to be secured by NHS England over the past five years, bringing significant benefit for patients – and thanks to funding through our Innovative Medicines Fund, this one-off therapy will be fast-tracked to patients who could benefit from the new lease of life it promises.”
Prof Bola Owolabi, the 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 are committed to reducing healthcare inequalities by rolling out new and pioneering treatments on the NHS for conditions, such as thalassaemia, which disproportionally affects people from some minority ethnic backgrounds.”
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Romaine Maharaj, the 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.
“It is a beacon of hope that underscores the power of innovation in medicine, paving the way for curative options that can truly enhance the quality of life for everyone affected.”