The European Medicines Agency recommended approval for the oral once-daily therapy Xospata (gilteritinib) as a monotherapy for the treatment of adult patients who have relapsed or refractory (resistant to treatment) acute myeloid leukemia with a FLT3 mutation (FLT3mut+).
The Committee for Medicinal Products for Human Use gave the positive opinion. If approved by the European Commission, Xospata has the potential to improve treatment outcomes for AML patients with the most common mutations – FLT3 internal tandem duplication and FLT3 tyrosine kinase domain–and would be one of the few advances for the treatment of AML in Europe over the past 40 years.
Xospata received accelerated assessment from the EMA, which allowed the CHMP to reduce the timeframe for approval. Astellas Pharma Inc. sponsors Xospata.
“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,” study investigator Giovanni Martinelli, Institute of Hematology, S.Orsola-Malpighi University Hospital, Bologna, Italy, said in a statement. “For relapsed or refractory FLT3mut+ AML patients the current prognosis is poor, with median OS of less than six months following treatment with salvage chemotherapy. If approved by the EC, gilteritinib has the potential to change the treatment landscape.”
The CHMP decision is based on results from the phase III ADMIRAL trial, which investigated Xospata versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. Patients treated with gilteritinib had significantly longer 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 Xospata, compared to 17% for patients who received salvage chemotherapy.
In late 2018, Xospata was approved by regulatory agencies in the United States and Japan for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML.
