FDA Approves Treatment for Acute Myeloid Leukemia

The FDA has approved midostaurin (Rydapt, Novartis) for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML), in combination with chemotherapy. The drug is indicated for patients who have a specific genetic mutation called FLT3 and is approved with a companion diagnostic, Invivoscribe Technologies’ LeukoStrat CDx FLT3 Mutation Assay, which is used to detect the FLT3 mutation.

If the FLT3 mutation is detected in the patients’ blood or bone marrow samples, then the patient may be eligible for treatment with Rydapt in combination with the chemotherapy regimen of standard cytarabine and daunorubicin induction and cytarabine consolidation.

The approval is based on a clinical trial that demonstrated Rydapt’s safety and efficacy for patients with AML. The study included 717 patients, who had not been previously treated for AML, who received Rydapt in combination with chemotherapy treatment. Patients who received Rydapt lived longer than patients who received chemotherapy alone, although a specific median survival rate could not be reliably estimated.

Patients who received Rydapt in the trial also went longer without certain complications than patients who received chemotherapy alone.

Common adverse effects include low levels of white blood cells with fever, nausea, inflammation of the mucous membranes, vomiting, headaches, spots on the skin due to bleeding, musculoskeletal pain, nosebleeds, device-related infection, high blood sugar, and upper respiratory tract infection.

Rydapt was also approved for adults with certain types of rare blood disorders, such as aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, and mast cell leukemia.

Reference

FDA approves new combination treatment for acute myeloid leukemia [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555778.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 28, 2017.