British regulators are set to support a gene therapy for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency, it has been announced.
The disorder affects around three babies a year in England and current treatment involves stem cell transplant to restore immune function.
Now, a new gene therapy treatment, Strimvelis, has gained backing from the National Institute for Health and Care Excellence in a consultation document issued last week.
The treatment could be used when no suitable stem cell donor is available, the guidance states. During treatment, the patient's bone marrow cells are altered, activating the ADA enzyme. It is only available from a centre in Milan, Italy, and costs 594,000 Euros for the single treatment.
NICE has been told that Strimvelis gives better overall survival than stem cell transplants – and is less risky.
Professor Carole Longson, from NICE, said: "Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of disadvantages of current treatments.
“This means that children born with ADA-SCID will now have a better chance of being able to lead as near normal a life as possible, going to school, mixing with friends, free from the constant threat of getting a potentially life-threatening infection.
"Our evaluation of Strimvelis is the first time that NICE has applied the higher cost effectiveness limits introduced last year for the assessment of treatments for these extremely rare conditions."
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