Gilteritinib receives positive recommendation from EMA CHMP for the treatment of FLT3-mutated AML

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended that gilteritinib is granted marketing authorization in Europe for the treatment of FLT3-mutated relapsed/refractory (R/R) acute myeloid leukemia (AML).1

Gilteritinib was previously approved for use in the United States (US) by the US Food and Drug Administration (FDA) in November 2018.2 The EMA CHMP undertook this review as part of the accelerated assessment procedure, designed for therapies of major public health interest.1

The approvals are based on the results of the phase III ADMIRAL study (NCT02421939), which is a randomized trial comparing the use of gilteritinib (120mg/day) to salvage chemotherapy (SC) in patients with FLT3-mutated R/R AML. In total, 138 patients were treated with gilteritinib or SC, with gilteritinib administered until unacceptable toxicity or lack of clinical benefit.2

Interim analysis from the study, with a median follow-up of 4.6 months, showed:2

  • Complete response (CR) / CR with partial hematologic recovery (CRh): 21% (n= 29)
  • Within any 56-day baseline period:
    • Of the 106 patients reliant on red blood cell (RBC) and/or platelet transfusions at baseline, 33 became independent of RBC/platelet transfusions
    • Of the 32 patients who were independent of both RBC and platelet transfusions at baseline, 17 remained independent
  • Most common adverse events (AEs) in ≥20% of patients:
    • Myalgia/arthralgia
    • Transaminase increase
    • Fatigue/malaise
    • Fever
    • Noninfectious diarrhea
    • Dyspnea
    • Edema
    • Rash
    • Pneumonia
    • Stomatitis
    • Cough
    • Headache
    • Hypotension
    • Dizziness
    • Vomiting
  1. European Medicines Agency. Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 16-19 September 2019. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-16-19-september-2019 [Accessed 2019 Sep 20]
  2. U.S. Food and Drug Administration. FDA approves gilteritinib for relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation. https://www.fda.gov/drugs/fda-approves-gilteritinib-relapsed-or-refractory-acute-myeloid-leukemia-aml-flt3-mutatation [Accessed 2019 Sep 20]

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