FDA Approves Gilteritinib for Relapsed or Refractory FLT3-Mutated AML


Today, the U.S. Food and Drug Administration (FDA) approved gilteritinib (Xospata) for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation as detected by an FDA-approved test.

The FDA also approved an expanded indication for a companion diagnostic to include use with gilteritinib. The LeukoStrat CDx FLT3 Mutation Assay is used to detect the FLT3 mutation in patients with AML.


Approval of gilteritinib was based on an interim analysis of the ADMIRAL trial, which included 138 adult patients with relapsed or refractory AML having a FLT3 internal tandem duplication (ITD), D835, or I836 mutation detected by the LeukoStrat CDx FLT3 Mutation Assay. Gilteritinib was given orally at a dose of 120 mg daily until unacceptable toxicity or lack of clinical benefit.

After a median follow-up of 4.6 months (range = 2.8–15.8 months), 29 patients achieved complete remission or complete remission with partial hematologic recovery (21%, 95% confidence interval [CI] = 14.5–28.8).

Among the 106 patients who were dependent on red blood cell and/or platelet transfusions at baseline, 33 (31.1%) became independent of red blood cell and platelet transfusions during any 56-day postbaseline period. For the 32 patients who were independent of both red blood cell and platelet transfusions at baseline, 17 (53.1%) remained transfusion-independent during any 56-day postbaseline period.

The most common adverse reactions occurring in ≥ 20% of patients were myalgia/arthralgia, transaminase increase, fatigue/malaise, fever, noninfectious diarrhea, dyspnea, edema, rash, pneumonia, nausea, stomatitis, cough, headache, hypotension, dizziness, and vomiting.

The recommended gilteritinib dose is 120 mg orally once daily.

View the prescribing information for gilteritinib.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.




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