Astellas Pharma : Receives Positive CHMP Opinion for XOSPATA® (gilteritinib) as a Monotherapy for Patients with Relapsed or Refractory Acute Myeloid Leukemia with a FLT3 Mutation

If approved by the European Commission, gilteritinib would represent one of
the few advances in Europe for AML over the past 40 years¹

TOKYO, Sept. 20, 2019 - Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., 'Astellas') announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending 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 (AML) with a FLT3 mutation (FLT3mut+).² If approved by the European Commission (EC), gilteritinib has the potential to improve treatment outcomes for AML patients with the most common mutations - FLT3 internal tandem duplication (ITD) and FLT3 tyrosine kinase domain (TKD) - and would be one of the few advances for the treatment of AML in Europe over the past 40 years.^1,3 Gilteritinib received accelerated assessment from the EMA, which allowed the CHMP to reduce the timeframe for approval.⁴

'The data are encouraging, showing a significant improvement in overall survival (OS), and one-year survival rates doubled when comparing gilteritinib to the current standard of care,' said Giovanni Martinelli, M.D., Institute of Hematology, S.Orsola-Malpighi University Hospital, Bologna, Italy, a study investigator. '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 3 ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML.⁵ Patients treated with gilteritinib had significantly longer OS than those who received salvage chemotherapy.⁵ 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).^5,6 Rates of one-year survival were 37% for patients who received gilteritinib, compared to 17% for patients who received salvage chemotherapy.⁵

'There is a high unmet need in AML and Astellas is committed to improving treatment options. Gilteritinib offers a potential new alternative for patients with relapsed or refractory FLT3mut+ AML, with data showing improved survival outcomes,' said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. 'Subject to EC approval, gilteritinib has the potential to provide new hope for clinicians, patients and their families.'

The positive opinion from the CHMP will now be reviewed by the EC, which has the authority to approve medicines for the 28 European Union member countries, and is also valid in Iceland, Norway and Liechtenstein.⁷ In late 2018, gilteritinib was approved by regulatory agencies in the U.S. and Japan for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML.^8,9

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