The European Commission (EC) has approved the oral once-daily therapy gilteritinib as a monotherapy for the treatment of adult patients with relapsed or refractory (resistant to treatment) acute myeloid leukaemia (AML) with a FLT3 mutation (FLT3mut ).

Gilteritinib has the potential to improve treatment outcomes for AML patients with two forms of the most common mutation - FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain (TKD) mutation.

This approval is based on results from the Phase 3 ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut AML.

Patients treated with gilteritinib had significantly longer overall survival (OS) than those who received salvage chemotherapy.

Median OS for patients who received gilteritinib was 9.3 months, compared to 5.6 months for patients who received salvage chemotherapy (Hazard Ratio = 0.64 (95% CI 0.49, 0.83), P=0.0004).

Rates of one-year survival were 37% for patients who received gilteritinib, compared to 17% for patients who received salvage chemotherapy.

“AML is a rare cancer and patients with a FLT3 mutation have a particularly poor prognosis, with a median survival of less than six months following treatment with salvage chemotherapy,” said Giovanni Martinelli, M.D., Institute of Hematology, S.Orsola-Malpighi University Hospital, Bologna, Italy, an investigator in the ADMIRAL trial. “Gilteritinib is a new and clinically meaningful treatment option that provides a welcome advance for patients and health care professionals across the European Union.”

The EC marketing authorisation for gilteritinib in relapsed or refractory FLT3mut AML is applicable to the European Union (EU) member countries, and is also valid in Iceland, Norway and Liechtenstein.

Gilteritinib has been designated an orphan medicinal product and also received accelerated assessment from the European Medicines Agency earlier this year, which reduced the timeframe for approval.

“Today’s approval marks a significant advance for patients living with relapsed or refractory, FLT3 mutation-positive acute myeloid leukaemia,” said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. “We look forward to working with health authorities across the EU to bring gilteritinib to patients who need it the most, as soon as possible.”

Patients’ FLT3mut status can change over the course of AML treatment, even after relapse.

Due to the poor outcomes associated with FLT3mut AML, patients’ FLT3 mutation status may be confirmed to help inform the best treatment approach.

Source: Astellas