People in England living with a rare genetic blood disorder are to be offered a new, ‘life-changing’ treatment through the NHS in what has been described as a historic moment.
Casgevy, a one-off gene therapy, has been approved for use by the NHS in England for people with severe thalassemia.
The treatment was approved on Wednesday by the National Institute for Health and Care Excellence (Nice), meaning around 460 patients currently suffering from transfusion-dependent beta thalassemia could be eligible for the treatment.
Thalassemia is the name for a group of inherited blood disorders in which the body does not produce enough haemoglobin. It affects around 2,300 people in the UK, who are mainly from the Mediterranean, Asia or the Middle East. Many people with beta thalassemia are not expected to live beyond the age of 50.
Casgevy works by editing a gene in the recipient’s bone marrow stem cells so that the body produces functioning hemoglobin.
The hope is that the therapy will be a lifelong cure. In international clinical trials, 93% of patients with beta thalassemia did not need a blood transfusion for at least a year after treatment.
Amanda Pritchard, NHS Chief Executive, said: “This is a historic moment for people living with beta thalassaemia, as a potential cure for those living with this debilitating condition becomes available through the NHS.
“Normally, patients experience painful side effects and undergo frequent blood transfusions, which seriously impacts their quality of life, but this therapy offers people a life without those side effects, and also the hope of living longer, which is really great news.
“This is the latest in a series of revolutionary gene therapies secured by NHS England over the past five years, bringing significant benefits to patients. And funding through our Innovative Medicines Fund means this one-off therapy can be delivered quickly to patients who can benefit from the new life expectancy it promises.”
Prof 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 dramatically change the lives of people with what can be an extremely painful condition.
“We are committed to reducing inequalities in healthcare by introducing new and ground-breaking treatments into the NHS for conditions such as thalassaemia, which disproportionately affects people from certain ethnic minority groups.”
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Romaine Maharaj, director of the British Thalassaemia Society, said: “Now that Nice has approved gene therapy for transfusion-dependent thalassaemia under the NHS managed access programme, we are on the cusp of a revolutionary breakthrough.
“This transformative treatment offers patients a life-changing opportunity, allowing 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 and paves the way for curative options that can truly improve the quality of life for everyone affected.”