Hundreds of people living with a rare genetic blood disorder are set to benefit from a ‘life-changing’ therapy on the NHS.
Casgevy, which is a one-off gene therapy, has been approved by the National Institute for Health and Care Excellence to treat people living with a severe form of thalassaemia.
Amanda Pritchard, the chief executive of the NHS called this a ‘historic moment’ for people living with beta thalassaemia, with the treatment offering a potential cure for those facing this debilitating disorder.
‘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,’ she said.
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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 patient’s bone marrow stem cells so that the body produces functioning haemoglobin. 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.
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.
‘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.’