Xospata (gilteritinib) tablets were approved today by the U.S. Food and Drug Administration for treatment of relapsed or refractory acute 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 Mutation Assay used to detect the FLT3mutation in AML patients.
About 25 to 30 percent of patients with AML have a mutation in the FLT3 gene, which is associated with a more aggressive form of AML and higher relapse risk, Richard Pazdur, M.D., director of the FDA Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research, was quoted as saying in the release.
Xospata, the first drug 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 confirmed FLT3 mutation. Researchers found that 21 percent of patients achieved complete remission or complete remission with partial hematologic recovery. One hundred six patients required red blood cell or platelet transfusion when they started treatment with Xospata; of these patients, 31 did not require transfusion for 56 days or longer after starting Xospata.
Commonly reported side effects of Xospata 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. The agency also warned that cases of differentiation syndrome have been reported, although rare. Pregnant women and those who are breastfeeding should not take Xospata.