Single-Agent FLT3 Inhibitor Gets FDA Nod for Acute Leukemia - MedPage Today
WASHINGTON -- The FDA approved gilteritinib (Xospata) on Wednesday as monotherapy for adults with FLT3-positive acute myeloid leukemia (AML) in the relapsed or refractory setting.
"These mutations are associated with a particularly aggressive form of the disease and a higher risk of relapse," Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence, said in the statement announcing the approval. "Xospata targets this gene and is the first drug to be approved that can be used alone in treating patients with AML having a FLT3 mutation who have relapsed or who don't respond to initial treatment."
The approval was based on results of ADMIRAL, a study of 138 relapsed or refractory AML patients with FLT3 internal tandem duplication D835 or I836 mutations treated with daily single-agent 120-mg gilteritinib. At a median follow-up at 4.6 months, treatment with gilteritinib yielded complete remissions (CRs), or CRs with partial hematologic recovery in 21% of patients (95% CI 14.5%-28.8%).
The need for red blood cell and/or platelet transfusions was also reduced. Among the 106 patients who were dependent on transfusions for either at baseline, 31.1% no longer required transfusions during the 56 days from the start of the trial. And, more than half (53.1%) of the 32 patients who were transfusion-independent at baseline, remained so at 56 days from the start of the trial.
In conjunction with the approval, the FDA expanded the indication of the LeukoStrat CDx FLT3 Mutation Assay -- which was used to detect FLT3 mutations in the trial, the test is now formally approved to identify patients eligible for gilteritinib.
Common adverse events (≥20%) in ADMIRAL included coughing, diarrhea, dizziness, dyspnea, edema, fatigue/malaise, fever, headache, hypotension, myalgia/arthralgia, nausea, transaminase increases, pneumonia, rash, stomatitis, and vomiting. The FDA also warned of rare cases of differentiation syndrome observed in patients on gilteritinib, and said that the drug should not be used for pregnant women or for those breastfeeding due to the potential of harm to the fetus or baby.
The FDA last year approved the FLT3 inhibitor midostaurin (Rydapt) in combination with chemotherapy for patients with FLT3-mutated AML. Another FLT3 inhibitor, quizartinib, has shown modest improvement in the treatment of AML in the salvage setting, and is under priority review by the FDA.
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