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Innovation Observatory > Reports > Drugs > Gilteritinib for Relapsed or Refractory Acute Myeloid Leukaemia (AML) with an FLT3 Mutation – Second Line

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Gilteritinib for Relapsed or Refractory Acute Myeloid Leukaemia (AML) with an FLT3 Mutation – Second Line


Cancer and Palliative Care

May 2018

Acute myeloid leukaemia (AML) is a type of cancer that causes the bone marrow to produce lots of immature white blood cells. It is most common in people aged 60 years and over. Symptoms of AML may include weakness, fatigue, shortness of breath, recurrent infections, prolonged bleeding, loss in appetite and unintended weight loss. Most patients with AML are treated with standard
chemotherapy. AML that is non‐responsive to treatment is called refractory while that which returns after response to initial treatment is called relapsed. Relapsed and refractory AML (RR‐AML) are associated with a poor prognosis. Mutations in a gene called FMS‐like tyrosine kinase (FLT3) is the most often present mutation in patients with AML. There are currently no approved targeted therapies specifically for RR‐AML with an FLT3 mutation. AML typically develops rapidly and is fatal unless treated.
Gilteritinib is a drug being developed for patients with RR‐AML with FLT3 mutation. It is an inhibitor of mutant‐FLT3 enzymes and acts by blocking the production of chemicals that cause the abnormal (cancerous) white blood cells to grow. Gilteritinib is given through the oral route once a day. As there are currently limited treatment options for patients with RR‐AML that have a FLT3 mutation, gilteritinib, if licensed, has the potential to offer a new treatment option for this group of patients.

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