The FDA has cleared midostaurin (Rydapt, Novartis) for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who have the FLT3 gene mutation, in combination with chemotherapy. The drug is approved for use with a companion diagnostic, the LeukoStrat CDx FLT3 mutation assay (Invivoscribe Technologies), which is used to detect the FLT3 mutation in patients with AML.
Midostaurin, a kinase inhibitor, blocks several enzymes that promote cell growth. If the FLT3 mutation is detected in blood or bone marrow samples using the LeukoStrat CDx FLT3 mutation assay, the patient may be eligible for treatment with midostaurin in combination with chemotherapy.
The safety and efficacy of midostaurin in AML were studied in a randomized trial of 717 patients who had not been treated for the disease. The patients who received midostaurin in combination with chemotherapy lived longer than those who received chemotherapy alone, although a specific median survival rate could not be reliably estimated. In addition, patients who received midostaurin in combination with chemotherapy went longer (median period, 8.2 months) without certain complications (failure to achieve complete remission within 60 days of starting treatment, and progression of leukemia or death) compared with patients who received chemotherapy alone (median period, 3.0 months).
Common adverse effects of midostaurin in patients with AML include febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infection.
Midostaurin was also approved for the treatment of adults with the following rare blood disorders: aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm, and mast-cell leukemia. Common adverse effects of midostaurin in these patients include nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, fatigue, upper respiratory tract infection, constipation, fever, headache, and shortness of breath.
Source: FDA; April 28, 2017.