FDA News

FDA approves Xospata for relapsed, refractory FLT3-mutated acute myeloid leukemia

The FDA today approved gilteritinib for the treatment of adults with relapsed or refractory acute myeloid leukemia and a FLT3 mutation as detected by an FDA-approved test.

The FDA also expanded the indication for a companion diagnostic — the LeukoStrat CDx FLT3 Mutation Assay (Invivoscribe Technologies) — to include use with gilteritinib (Xospata, Astellas Pharma) to detect the FLT3 mutation in patients with AML.

The approval of gileritinib was based, in part, on data from an interim analysis of the ADMIRAL trial. The study included 138 adults with relapsed or refractory AML who harbored a FLT3 ITD, D835 or I836 mutation.

Patients received 120 mg daily gilteritinib until unacceptable toxicity or lack of clinical benefit.

Median follow-up was 4.6 months (range, 2.8-15.8).

Twenty-nine patients (21%; 95% CI, 14.5-28.8) achieved complete remission or complete remission with partial hematologic recovery.

In total, 33 of 106 patients (31.1%) who were dependent on red blood cell and/or platelet transfusions at baseline became independent of these transfusions during any 56-day post-baseline period.

Common adverse events that occurred in 20% or more of the study population included myalgia/arthralgia, transaminase increase, fatigue/malaise, fever, noninfectious diarrhea, dyspnea, edema, rash, pneumonia, nausea, stomatitis, cough, headache, hypotension, dizziness and vomiting.

As HemOnc Today previously reported, FDA granted this application fast track and priority review, as well as orphan product designation.


The FDA today approved gilteritinib for the treatment of adults with relapsed or refractory acute myeloid leukemia and a FLT3 mutation as detected by an FDA-approved test.

The FDA also expanded the indication for a companion diagnostic — the LeukoStrat CDx FLT3 Mutation Assay (Invivoscribe Technologies) — to include use with gilteritinib (Xospata, Astellas Pharma) to detect the FLT3 mutation in patients with AML.

The approval of gileritinib was based, in part, on data from an interim analysis of the ADMIRAL trial. The study included 138 adults with relapsed or refractory AML who harbored a FLT3 ITD, D835 or I836 mutation.

Patients received 120 mg daily gilteritinib until unacceptable toxicity or lack of clinical benefit.

Median follow-up was 4.6 months (range, 2.8-15.8).

Twenty-nine patients (21%; 95% CI, 14.5-28.8) achieved complete remission or complete remission with partial hematologic recovery.

In total, 33 of 106 patients (31.1%) who were dependent on red blood cell and/or platelet transfusions at baseline became independent of these transfusions during any 56-day post-baseline period.

Common adverse events that occurred in 20% or more of the study population included myalgia/arthralgia, transaminase increase, fatigue/malaise, fever, noninfectious diarrhea, dyspnea, edema, rash, pneumonia, nausea, stomatitis, cough, headache, hypotension, dizziness and vomiting.

As HemOnc Today previously reported, FDA granted this application fast track and priority review, as well as orphan product designation.