The drug’s application was designated for fast track and priority review, as well as orphan product status.
The FDA today approved gilteritinib (Xospata, Astellas Pharma US Inc.) for treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, as detected by an agency-approved test. The drug’s application had been granted fast track and priority review, as well as orphan product designation, by the FDA.
The FDA also approved an expanded indication for a companion diagnostic, to include use with gilteritinib. The LeukoStrat CDx FLT3 Mutation Assay, developed by Invivoscribe Technologies, Inc., is used to detect the FLT3 mutation in patients with AML.
Approval of gilteritinib was based on an interim analysis of the ADMIRAL trial (NCT02421939), which included 138 adult patients with relapsed or refractory AML having a FLT3 ITD, D835, or I836 mutation 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 to 15.8), 29 patients achieved complete remission (CR) or CR with partial hematologic recovery (CRh) (21%, 95% CI: 14.5, 28.8).
Among the 106 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 33 (31.1%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. For the 32 patients who were independent of both RBC and platelet transfusions at baseline, 17 (53.1%) remained transfusion-independent during any 56-day post-baseline 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.
FDA approves gilteritinib for relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation [news release]. Silver Spring, MD; November 28, 2018: FDA website. http://www.pharmacytimes.com/link/236. Accessed November 28, 2018.