Additional treatment for AML patients now available in England

Webpage last updated on: 19th September 2024

What’s the news?

Today, Leukaemia Care welcomes the decision by the National Institute for Healh and Care Excellence (NICE) to approve quizartinib (Vanflyta©) for use on the NHS in Wales and England, with Northern Ireland likely to follow this decision too. This is a treatment for patients with newly diagnosed acute myeloid leukaemia (AML) with an FMS-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutation. Quizartinib can be used in combination with chemotherapy during induction and consolidations treatment, followed by quizartinib treatment on its own during maintenance treatment.

Quizartinib is not current available on the NHS in Scotland but is currently under assessment. We will announce if it becomes available here too.

Leukaemia Care submitted a written response to NICE representing the views of AML patients. A submission is created by drawing on experiences of individual patients and data from our surveys. We also nominated a patient, Esther Beswick, to speak about the experience of AML patients at the committee meeting for the NICE decision. Esther described problems with current treatments from the viewpoint of AML patients.

Leukaemia Care’s CEO, Colin Dyer, said of today’s decision:

“We welcome the decision by NICE to approve this treatment, and are pleased that both parties were able to reach an agreement without further delay in access for patients. Patients with FLT3-ITD mutations have an increased risk of relapse with standard treatments, so another targeted treatment option for such patients is important for clinicians to tailor treatment plans to their patients effectively.”

Why is this news important for AML patients with an FLT3-ITD mutation?

In newly diagnosed AML, the preferred primary induction treatment is intensive chemotherapy. However, this treatment can be ineffective for patients. FLT3-ITD mutated AML often can be put into remission by chemotherapy alone, but often relapses later on. Clinicians consulted as part of the NICE appraisal stated they would never give these patients chemotherapy alone because of this. The main clinical trial of quizartinib that was examined in the process should a more than doubling of the average survival time (overall survival) between people having quizartinib compared to people having just chemotherapy.

There are other treatments that can be used alongside chemotherapy to boost the response of FLT3-ITD mutated AML. The main one approved by NICE is midostaurin, which also works against cells that have other mutation in the FLT3 gene. However, quizartinib targets the mutated cell proteins more specifically than other treatments, and so is a useful alternative option for patients and their clinical teams to consider. Unlike other treatments for this group of mutations, quizartinib is also an option for maintenance treatment of patients who have a stem cell transplant after their consolidation treatment. Maintenance treatment is an important way that haematologists ensure that the treatment given for aggressive leukaemia is long lasting.

How is the treatment administered?

Quizartinib is an oral tablet treatment that is taken alongside standard chemotherapy. The chemotherapy is given intravenously. Quizartinib will be given throughout the induction, consolidation and maintenance phases of treatment and doses may vary over the course of treatment.

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