FDA approves Rydapt in combination with Chemotherapy for treatment of AML

The U.S. FDA approved Rydapt (midostaurin) in combination with Chemotherapy for the treatment of adult patients with newly diagnosed AML (acute myeloid leukemia) who have a specific genetic mutation called FLT3. The drug is approved for use with a companion diagnostic, the LeukoStrat CDx FLT3 Mutation Assay, which is used to detect the FLT3 mutation in patients with AML.

Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence, said that Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy. The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment.

In the trial, patients who received Rydapt in combination with chemotherapy lived longer in the trial went longer (median 8.2 months) without certain complications  than patients who received chemotherapy alone.

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), device-related 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.