Quizartinib Recommended for Approval in EU by CHMP for Patients with Newly Diagnosed FLT3-ITD Positive AML

16 September 2023

Daiichi Sankyo (TSE: 4568) has announced that quizartinib has received a favorable recommendation for approval in the European Union (EU). This approval is for its use in combination with standard cytarabine and anthracycline induction, as well as standard cytarabine consolidation chemotherapy. Following these treatments, patients can continue with quizartinib as a single-agent maintenance therapy. This recommendation applies to adult patients who have newly diagnosed acute myeloid leukemia (AML) and test positive for FLT3-ITD.

The positive recommendation comes from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Their decision is based on the results of the phase 3 QuANTUM-First trial, which were published in The Lancet. The recommendation will now undergo review by the European Commission, the body responsible for granting marketing authorizations for medicines in the EU.

In the QuANTUM-First trial, quizartinib, when used in combination with standard induction and consolidation therapies, followed by maintenance therapy, demonstrated a 22% reduction in the risk of death compared to standard chemotherapy alone. This was especially significant for patients with newly diagnosed FLT3-ITD positive AML. The median overall survival for patients receiving quizartinib was 31.9 months, compared to 15.1 months for patients in the control group, at a median follow-up of 39.2 months.

Mark Rutstein, MD, Global Head of Oncology Clinical Development at Daiichi Sankyo, stated, "Today’s positive CHMP opinion for quizartinib is an important step towards translating the clinical benefit observed in QuANTUM-First into an approved treatment option for patients in the EU with the difficult-to-treat FLT3-ITD subtype of acute myeloid leukemia. If approved, quizartinib would be the first FLT3 inhibitor approved specifically for patients with newly diagnosed FLT3-ITD positive AML."

The safety profile of quizartinib in the trial was consistent with previous clinical trials, with no new safety concerns identified. The most common grade 3 or 4 treatment-related adverse events included febrile neutropenia (43%), hypokalemia (19%), neutropenia (18%), and pneumonia (11%). QTcF > 500 ms occurred in 2.3% of patients receiving quizartinib, and 0.8% of patients discontinued quizartinib due to QT prolongation. Ventricular arrhythmia events with quizartinib were rare, with two patients (0.8%) experiencing cardiac arrest with recorded ventricular fibrillation on ECG, one of which had a fatal outcome, both in the context of severe hypokalemia.