Gilteritinib (Xospata), a treatment for acute myeloid leukaemia (AML) with a FLT3 mutation, has received a positive opinion recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

If approved by the European Commission, gilteritinib has the potential to improve treatment outcomes for AML patients with the most common mutations (FLT3 internal tandem duplication positive/tyrosine kinase domain), and will be one of few advances for the treatment of AML in Europe over the past 40 years.

Gilteritinib received accelerated assessment from the EMA, which allowed the CHMP to reduce the timeframe for approval from 210 to 150 days.

Giovanni Martinelli, Institute of Hematology, S.Orsola-Malpighi University Hospital, Bologna, Italy, and a study investigator, said: “The data are encouraging, showing a significant improvement in overall survival, and one-year survival rates doubled when comparing gilteritinib to the current standard of care.

"For relapsed or refractory FLT3 mutation+ AML patients the current prognosis is poor, with median overall survival of less than six months following treatment with salvage chemotherapy.”

The CHMP decision is based on results from the Phase 3 ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3 mutation AML. Patients treated with gilteritinib had significantly longer overall survival than those who received salvage chemotherapy. Median overall survival 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 37.1% for patients who received gilteritinib, compared to 16.7% for patients who received salvage chemotherapy.