AML gilteritinib treatment approved by the European Commission

The European Commission (EC) has approved Astellas Pharma Inc’s oral once-daily therapy Xospata™ (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, the company has said.

“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, MD, Institute of Hematology, S.Orsola-Malpighi University Hospital. “Gilteritinib is a new and clinically meaningful treatment option that provides a welcome advance for patients and healthcare professionals across the EU.” 

This approval is based on results from the Phase III 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. Rates of one-year survival were 37 percent for patients who received gilteritinib, compared to 17 percent for patients who received salvage chemotherapy.

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