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