FDA Approves Treatment for AML Patients With Gene Mutation

Xospata (gilteritinib) tablets were approved today by the You. S. Food and Medication Administration for treatment of relapsed or refractory serious myeloid leukemia (AML) in adults with an FLT3 mutation, the agency announced in a news release. The FDA also granted expanded approval to the LeukoStrat CDx FLT3 Veränderung Assay used to identify the FLT3 mutation in AML patients.

About twenty five to 30 % of patients with AML have a mutation in the FLT3 gene, which is of a more aggressive form of AML and higher relapse risk, Richard Pazdur, M. D., director of the FDA Oncology Centre of Excellence and behaving director of the Business office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research, was cited as saying in the release.

Xospata, the initial medication approved for treating this specific group of patients who do not respond to initial treatment, was evaluated in a clinical trial involving 138 patients with relapsed or refractory AML and a proved FLT3 mutation. Researchers found that 21 percent of patients achieved a complete remission or complete remission with partial hematologic recovery. 1 hundred six patients required red blood cell or platelet transfusion when they started treatment with Xospata; of these patients, 23 did not require transfusion for 56 days or longer after starting Xospata.

Commonly reported side outcomes of Xospata to include myalgia/arthralgia, fatigue, and liver transaminase. The agency advises health care providers to monitor patients for posterior reversible encephalopathy syndrome, prolonged QT interval, and pancreatitis. Typically the agency also warned that cases of differentiation syndrome have been reported, although rare. Pregnant women and those who are nursing should not take Xospata.