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Gilteritinib becomes first NICE-approved oral therapy for relapsed or refractory acute myeloid leukaemia

Published on 16/07/20 at 10:22am

NICE has revealed its recommendation of Astellas’ gilteritinib for routine use as a monotherapy on the NHS in England and Wales for the treatment of relapsed or refractory FLT3 mutation-positive acute myeloid leukaemia (AML) in adult patients.

The decision makes the therapy the first oral monotherapy approved in this indication in the UK, allowing patients to medicate at home rather than in a hospital, and reverses the Institute’s ruling in January this year that the therapy could not be considered a cost-effective use of NHS resources.  

With the submission of supplementary clinical data showing that gilteritinib demonstrated superior overall survival benefit compared to chemotherapy, increasing median OS from 5.6 months to 9.3 months, NICE could finally consider the therapy cost-effective.

However, despite the recommendation, NICE could not support the use of the drug as a maintenance therapy following a haematopoietic stem cell transplant, due to “considerable uncertainty about long-term survival”.

Patients with relapsed or refractory AML have limited options for treatment, with five-year survival rates for men and women standing at just 15% and 16% respectively. While salvage chemotherapy is the standard of care, this is an intensive course of treatment with a range of side-effects that can impair quality of life. An estimated 443 patients are thought to be eligible to receive the therapy.

Matt Fellows

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