Quizartinib Approved for First-Line Treatment of FLT3-ITD Positive Acute Myeloid Leukemia in Adults by the MHRA

The Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom has granted marketing authorization for Quizartinib (Vanflyta) as first-line therapy in conjunction with standard chemotherapy for adult patients diagnosed with FLT3-ITD positive acute myeloid leukemia (AML). This decision signifies a significant advancement in the treatment landscape for this specific subgroup of AML patients.

Acute Myeloid Leukemia and FLT3-ITD Mutation

AML is an aggressive hematologic malignancy characterized by uncontrolled proliferation of abnormal myeloid precursors in the bone marrow, leading to disruption of healthy blood cell production. The FLT3-ITD mutation, present in a subset of AML patients, contributes to a more aggressive disease course.

Mechanism of Action of Quizartinib

Quizartinib functions as a targeted therapy by inhibiting the activity of tyrosine kinases. These enzymes play a pivotal role in signal transduction pathways that promote the proliferation and survival of abnormal AML cells harboring the FLT3-ITD mutation. By blocking these pathways, Quizartinib disrupts the malignant growth cycle of these cells.

Clinical Trial Data Supporting Approval

The MHRA’s approval is based on robust clinical trial data from a randomized, double-blind, placebo-controlled Phase 3 study. This trial enrolled over 500 adult patients with newly diagnosed FLT3-ITD positive AML. The study demonstrated a statistically significant improvement in overall survival for patients receiving Quizartinib combined with chemotherapy compared to those receiving placebo with chemotherapy.

Safety Considerations and Continued Monitoring

As with all medications, Quizartinib can cause adverse events. The MHRA emphasizes the importance of ongoing safety monitoring to ensure patient well-being. Healthcare professionals are encouraged to report any suspected side effects associated with Quizartinib.


The MHRA’s approval of Quizartinib represents a major step forward in the management of FLT3-ITD positive AML. This targeted therapy offers a promising new treatment option for this subset of patients, potentially leading to improved clinical outcomes.

Posted March 2024.


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