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Gene therapy costing over £1.6m is approved for NHS patients with inherited blood disorder | UK News
The medicines watchdog is allowing the treatment on the NHS - after securing a discount on the normal price - and says it "could represent a potential cure" for people with a condition called beta-thalassaemia.
The National Institute for Health and Care Excellence (NICE) said in new final draft guidance that Casgevy, also known as exa-cel, will immediately be made available on the NHS to treat severe beta-thalassaemia while further evidence is collected on its cost-effectiveness. Helen Knight, director of medicines evaluation at NICE, said: "Although there are some uncertainties in the evidence for its long-term benefits, the committee felt exa-cel could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions." "Casgevy offers an effective cure for transfusion-dependent beta thalassaemia - a debilitating condition that was previously incurable in patients who don't have a stem cell donor."
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