NHS England has become the first health service in Europe to routinely commission quizartinib, a life-extending daily tablet, for patients newly diagnosed with acute myeloid leukemia (AML) who have the FLT3-ITD mutation. This fast-tracked approval, facilitated by the Cancer Drugs Fund, makes the treatment immediately available to eligible patients.
Clinical Impact of Quizartinib
Quizartinib, a targeted treatment, is designed to improve remission rates and long-term survival in AML patients with the FLT3-ITD mutation. This mutation affects approximately 27% of the 3,100 individuals diagnosed with AML in the UK each year. The treatment involves a once-daily tablet administered alongside chemotherapy during initial treatment, followed by up to three years as a maintenance therapy to reduce the risk of relapse.
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has determined the safety and effectiveness of quizartinib. Clinical trials showed that patients receiving quizartinib had a median overall survival of 31.9 months, compared to 15.1 months for those receiving a placebo. If a patient's condition improves with quizartinib and chemotherapy, they may become eligible for stem cell transplants, further enhancing their chances of being cured.
Mechanism of Action
AML with the FLT3-ITD mutation is characterized by uncontrolled cell growth within the bone marrow. Quizartinib functions as an inhibitor drug, specifically targeting and restricting the FLT3 enzyme responsible for this uncontrolled growth.
Expert Commentary
Professor Peter Johnson, NHS National Clinical Director for Cancer, stated, "It’s very good news that patients with this type of leukaemia now have an option that helps their chemotherapy to work better, boosting their chance of remission and long-term survival and offering them precious extra time with their families and friends."
Haran Maheson, Head of UK Oncology and Vice President at Daiichi Sankyo UK, noted, "Quizartinib with chemotherapy has been shown to have an improvement in median overall survival of 16.8 months compared to chemotherapy alone in a recent clinical study."
NICE's Perspective
Helen Knight, Director of Medicines Evaluation at NICE, commented, "Clinical evidence found patients taking quizartinib had an average overall survival of almost 32 months compared to just over 15 months for those receiving the placebo, and our independent committee found it to be a cost-effective treatment."
