FDA Approves Rydapt (Midostaurin) for Acute Myeloid Leukemia

Written By:Stacy Simon

The US Food and Drug Administration (FDA) has approved the first targeted therapy to treat adults with acute myeloid leukemia (AML). Rydapt (midostaurin) is intended to be used along with chemotherapy to treat newly diagnosed adults who have a mutation in a gene called FLT3. The FDA also approved a test to check for the mutation.

Rydapt is a type of targeted therapy called a kinase inhibitor. It works by blocking several proteins (kinases) on cancer cells, including FLT3, that can help the cells grow.

The FDA based its approval on a clinical trial of 717 people with AML who had not been previously treated. Those who received both Rydapt and chemotherapy lived longer than those who received only chemotherapy. In addition, those who received Rydapt and chemotherapy went longer (an average of 8.2 months) without certain complications than those who received chemotherapy alone (an average of 3 months).

The FDA also approved Rydapt for adults with the following blood disorders:  aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, and mast cell leukemia. Rydapt was evaluated using fast track, priority review, and breakthrough therapy designations – programs designed to speed up the availability of drugs to treat serious diseases.

Common side effects of Rydapt in people with AML include low levels of white blood cells with fever, nausea and vomiting, inflammation of the mucous membranes, headache, spots on the skin due to bleeding, muscle and bone pain, nosebleeds, infection, and high blood sugar.

Common side effects of Rydapt in people with rare blood disorders include nausea and vomiting, diarrhea, swelling, muscle and bone pain, abdominal pain, fatigue, upper respiratory tract infection, constipation, fever, headache, and shortness of breath.

Women who are pregnant or breastfeeding should not take Rydapt because it may cause harm to a developing fetus or a newborn baby.

Rydapt is marketed by Novartis Pharmaceuticals Corporation, and the FLT3 test is marketed by Invivoscribe Technologies, Inc.

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