"The U.S. Food and Drug Administration today approved Rydapt midostaurin for the treatment of adult patients with newly diagnosed acute myeloid leukemia AML who have a specific genetic mutation called FLT in combination with chemotherapy. The drug is approved for use with a companion diagnostic the LeukoStrat CDx FLT Mutation Assay which is used to detect the FLT mutation in patients with AML.   AML is a rapidly progressing cancer that forms in the bone marrow and results in an increased number of white blood cells in the bloodstream. The National Cancer Institute estimated that approximately  people would be diagnosed with AML in  and  were projected to die of the disease. Rydapt is the first targeted therapy to treat patients with AML in combination with chemotherapy said Richard Pazdur M.D. acting director of the Office of Hematology and Oncology Products in the FDAs Center for Drug Evaluation and Research and director of the FDAs Oncology Center of Excellence. The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment. Rydapt is a kinase inhibitor that works by blocking several enzymes that promote cell growth. If the FLT mutation is detected in blood or bone marrow samples using the LeukoStrat CDx FLT Mutation Assay the patient may be eligible for treatment with Rydapt in combination with chemotherapy. The safety and efficacy of Rydapt for patients with AML were studied in a randomized trial of  patients who had not been treated previously for AML. In the trial patients who received Rydapt in combination with chemotherapy lived longer than patients who received chemotherapy alone although a specific median survival rate could not be reliably estimated. In addition patients who received Rydapt in combination with chemotherapy in the trial went longer median . months without certain complications failure to achieve complete remission within  days of starting treatment progression of leukemia or death than patients who received chemotherapy alone median three months. Common side effects of Rydapt in patients with AML include low levels of white blood cells with fever febrile neutropenia nausea inflammation of the mucous membranes mucositis vomiting headache spots on the skin due to bleeding petechiae musculoskeletal pain nosebleeds epistaxis devicerelated infection high blood sugar hyperglycemia and upper respiratory tract infection. Rydapt should not be used in patients with hypersensitivity to midostaurin or other ingredients in Rydapt. Women who are pregnant or breastfeeding should not take Rydapt because it may cause harm to a developing fetus or a newborn baby. Patients who experience signs or symptoms of lung damage pulmonary toxicity should stop using Rydapt. Rydapt was also approved today for adults with certain types of rare blood disorders aggressive systemic mastocytosis systemic mastocytosis with associated hematological neoplasm or mast cell leukemia. Common side effects of Rydapt in these patients include nausea vomiting diarrhea swelling edema musculoskeletal pain abdominal pain fatigue upper respiratory tract infection constipation fever headache and shortness of breath. The FDA granted this application Priority Review https Fast Track https for the mastocytosis indication and Breakthrough Therapy https for the AML indication designations. The FDA granted the approval of Rydapt to Novartis Pharmaceuticals Corporation. The FDA granted the approval of the LeukoStrat CDx FLT Mutation Assay to Invivoscribe Technologies Inc. The FDA an agency within the U.S. Department of Health and Human Services protects the public health by assuring the safety effectiveness and security of human and veterinary drugs vaccines and other biological products for human use and medical devices. The agency also is responsible for the safety and security of our nations food supply cosmetics dietary supplements products that give off electronic radiation and for regulating tobacco products. Related Information   FDA Office of Hematology and Oncology Products https  FDA Approved Drugs Questions and Answers https  FDA Fast Track Breakthrough Therapy Accelerated Approval Priority Review https  FDA Companion Diagnostics https  NCI Adult AML https  GARD Systemic mastocytosis httpsrarediseases.info.nih.govdiseasessystemicmastocytosis"