The FDA ( U.S. Food and Drug Administration ) has approved Xospata ( Gilteritinib ) tablets for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia ( AML ) with a FLT3 mutation as detected by an FDA-approved test.
The FDA has also approved an expanded indication for a companion diagnostic, to include use with Xospata.
The LeukoStrat CDx FLT3 Mutation Assay is used to detect the FLT3 mutation in patients with acute myeloid leukemia.
Approximately 25 to 30% of patients with acute myeloid leukemia have a mutation in the FLT3 gene.
These mutations are associated with a particularly aggressive form of the disease and a higher risk of relapse.
Xospata targets this gene and is the first drug to be approved that can be used alone in treating patients with acute myeloid leukemia having a FLT3 mutation who have relapsed or who don’t respond to initial treatment.
Acute myeloid leukemia is a rapidly progressing cancer that crowds out normal cells in the bone marrow and bloodstream, resulting in low numbers of normal blood cells and a continuous need for transfusions.
The National Cancer Institute ( NCI ) estimates that approximately 19,520 people will be diagnosed with acute myeloid leukemia this year; approximately 10,670 patients with acute myeloid leukemia will die of the disease in 2018.
The efficiency of Xospata was studied in a clinical trial of 138 patients with relapsed or refractory acute myeloid leukemia having a confirmed FLT3 mutation.
Twenty-one percent of patients has achieved complete remission ( no evidence of disease and full recovery of blood counts ) or complete remission with partial hematologic recovery ( no evidence of disease and partial recovery of blood counts ) with treatment.
Of the 106 patients who required red blood cell or platelet transfusions at the start of treatment with Xospata, 31% became transfusion-free for at least 56 days.
Common side effects reported by patients in clinical trials were muscle and joint pain ( myalgia / arthralgia ), fatigue and elevated liver enzymes ( liver transaminase ).
Health care providers are advised to monitor patients for posterior reversible encephalopathy syndrome ( a syndrome characterized by headache, confusion, seizures and visual loss ), prolonged QT interval ( a heart rhythm condition that can potentially cause fast, chaotic heartbeats ) and pancreatitis ( inflammation in the pancreas ).
Rare cases of differentiation syndrome ( symptoms of which may include fever, cough, trouble breathing, fluid around the lungs or heart, rapid weight gain, swelling, and renal or hepatic dysfunction ) have been seen in patients taking Xospata.
Women who are pregnant or breastfeeding should not take Xospata because it may cause harm to a developing fetus or newborn baby. ( Xagena )
Source: FDA, 2018