Thalassaemia: NICE recommends “life changing” gene editing therapy

The National Institute for Health and Care Excellence (NICE) has recommended exagamglogene autotemcel (known as exa-cel, branded as Casgevy), a one off gene therapy treatment for severe β thalassaemia, in draft guidance.1NICE has recommended the therapy for patients with the inherited blood disorder who are aged 12 and over, need regular blood transfusions to manage their condition, and for whom no blood or bone marrow transplant donor is available. Exa-cel is the first medicine using the gene editing tool Crispr to be licensed for use in patients.2It is listed as costing £1.65m per course of treatment and will be made available on the NHS through the Innovative Medicines Fund. Treatment will be provided through a “managed access” agreement, with the therapy being rolled out to up to 460 eligible patients while more data on the benefits are gathered over the next five years.This comes after NICE rejected exa-cel for the…
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