Daiichi Sankyo’s Vanflyta (quizartinib) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) as a first-line treatment, alongside chemotherapy, for adults with acute myeloid leukaemia (AML) with a mutation in the FLT3 gene, FLT3-ITD.
The decision was based on evidence from the randomised, double-blind phase 3 clinical trial, which involved 539 newly diagnosed adult patients with FLT3-ITD-positive AML.
Responsible for around 3,100 new cases every year in the UK, AML is an aggressive, quick-progressing blood cancer that affects different types of blood cells in bone marrow, leading to bleeding, anaemia and infections.
According to the National Institute of Health, FLT3-ITD-positive AML occurs in approximately 20% of all AML cases.
The QuANTUM-First trial randomised patients to either receive Vanflyta or a placebo once daily for two weeks in each 28-day treatment cycle in combination with standard chemotherapy, followed by either Vanflyta or placebo once daily for up to 36 cycles of 28 days.
The trial demonstrated a statistically significant improvement in overall survival for patients receiving Vanflyta. Approximately 268 patients who received Vanflyta had a median overall survival of 31.9 months, compared to 15.1 months for the 271 patients receiving the placebo.
Julian Beach, interim executive director, healthcare quality and access, MHRA said: “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.
“As with all products, we will keep the safety of this medicine under close review.”
Taken orally once a day for two weeks during each cycle of chemotherapy, Vanflyta works to block a protein known as tyrosine kinases, to slow down or stop the production of abnormal bone marrow cells that do not mature into healthy cells, and to promote immature cell growth into normal cells.
In November 2023, the European Commission approved Vanflyta for adult patients with newly diagnosed AML that is FLT3-ITD positive in combination with standard chemotherapy followed by Vanflyta single-agent maintenance therapy.
The approval marked Vanflyta as the first FLT3 inhibitor to be approved in the EU.