New Life-Changing Treatment for Rare Blood Disorder Approved by NHS
People living with a severe form of thalassaemia in England will now have access to a potentially life-changing new treatment on the NHS, marking a historic moment in medical care.
Casgevy, a groundbreaking one-off gene therapy, has been approved by the National Institute for Health and Care Excellence (NICE) for use on the NHS. This treatment is specifically designed for individuals with a severe form of beta thalassaemia, a rare genetic blood disorder. Approximately 460 patients in England who currently rely on regular blood transfusions for their condition may be eligible for this new therapy.
Thalassaemia is a group of inherited blood disorders characterized by insufficient production of haemoglobin. It affects around 2,300 people in the UK, predominantly those of Mediterranean, Asian, or Middle Eastern descent. Many individuals with beta thalassaemia do not survive beyond their 50s.
How Casgevy Works
Casgevy addresses the root cause of beta thalassaemia by editing a gene in the patient’s bone marrow stem cells, allowing the body to produce functional haemoglobin. The treatment is hoped to be a lifelong cure. Clinical trials have shown that 93% of patients with beta thalassaemia who received Casgevy did not require a blood transfusion for at least a year following treatment.
Amanda Pritchard, NHS Chief Executive, described the approval as a historic achievement for those affected by beta thalassaemia. She highlighted that the treatment offers a potential cure and the possibility of a life free from the debilitating effects of regular blood transfusions. Pritchard also noted that this therapy is part of a series of revolutionary gene therapies secured by NHS England, funded through the Innovative Medicines Fund.
Prof. Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England, emphasized that this development represents a significant advance in the treatment of thalassaemia. He stressed the NHS’s commitment to reducing healthcare inequalities by providing pioneering treatments for conditions that disproportionately affect minority ethnic groups.
Romaine Maharaj, Executive Director of the UK Thalassaemia Society, praised the approval of Casgevy as a revolutionary breakthrough. Maharaj highlighted that the gene therapy offers patients a transformative opportunity to overcome their condition and embrace a future free from its challenges. This approval underscores the power of medical innovation and the potential for curative treatments to greatly enhance the quality of life for those affected.
This landmark decision represents a significant advancement in the treatment of thalassaemia, offering renewed hope and improved prospects for many patients.
