The UK is to have access to its first gene editing therapy for sickle cell disease and β-thalassaemia, it has been announced.
The CRISPR-based treatment, Casgevy, gained a licence from the Medicines and Healthcare products Regulatory Agency (MHRA) last week following a successful trial in the UK.
The treatment – which edits faulty genes in the patient’s bone marrow stem cells – will still need funding and may need assessment from the National Institute for Health and Care Excellence (NICE).
Some 29 sickle cell patients have completed a trial of the treatment – of which all but one reported freedom from severe pain crises for at least 12 months after treatment.
42 patients with β-thalassaemia have completed the trial – and 39 did not receive a red blood cell transfusion for at least 12 months. Researchers say side-effects are similar to those associated with autologous stem cell transplants.
John James, chief executive of the Sickle Cell Society, said: “There are limited medicines currently available to patients, so I welcome today’s news that a new treatment has been judged safe and effective, which has the potential to significantly improve the quality of life for so many.”
Julian Beach, from the MHRA, said: “Both sickle cell disease and β-thalassemia are painful, life-long conditions that in some cases can be fatal. To date, a bone marrow transplant – which must come from a closely matched donor and carries a risk of rejection – has been the only permanent treatment option.
“I am pleased to announce that we have authorised an innovative and first-of-its-kind gene-editing treatment called Casgevy, which in trials has been found to restore healthy haemoglobin production in the majority of participants with sickle-cell disease and transfusion-dependent β-thalassaemia, relieving the symptoms of disease.
“The MHRA will continue to closely monitor the safety and effectiveness of Casgevy, through real-world safety data and post-authorisation safety studies being carried out by the manufacturer.”
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