NICE recommends new gene therapy for adults with haemophilia B

The gene therapy Hemgenix (etranacogene dezaparvovec) should be made available on the NHS as a treatment option for adults with moderately severe or severe haemophilia B, the UK National Institute of Health and Care Excellence has recommended. NICE’s final draft guidance recommends the drug for NHS use with managed access over a limited five year period while further clinical evidence of its effectiveness is gathered.1Hemgenix is given by a one-off infusion over 1-2 hours. Currently, treatment with factor IX is repeated regularly, and people with severe haemophilia B typically need infusions once or twice a week. Other gene therapies already available on the NHS include Zolgensma for spinal muscular atrophy and Libmeldy for metachromatic leukodystrophy (MLD).23NICE initially rejected Hemgenix last year because of uncertainties in the long term clinical evidence and some of the assumptions used to estimate its cost effectiveness.4 But the watchdog has now shifted its recommendation after…
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