Rydapt First Drug in Decades Approved by the FDA for Acute Myeloid Leukemia

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On April 28, 2017, midostaurin (Rydapt; Novartis), a kinase inhibitor, became the first targeted therapy, and the first new drug in more than 20 years, to receive FDA approval, in combination with chemotherapy, for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) plus the FLT3 mutation.

Midostaurin was approved with a companion diagnostic test, the Leuko­Strat CDx FLT3 Mutation Assay, which detects the FLT3 mutation.

“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,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence.

On the same day, the FDA also approved Rydapt for the treatment of adults with other types of rare blood disorders, including aggressive systemic mastocytosis, systemic mastocytosis with associated hematologic neoplasm, or mast-cell leukemia.

Midostaurin was approved based on results of a randomized clinical trial of 717 patients with newly diagnosed AML who received midostaurin plus chemotherapy or placebo plus chemotherapy. Patients who received midostaurin plus chemotherapy had superior overall survival (OS) compared with those receiving placebo plus chemotherapy (hazard ratio [HR], 0.77; 2-sided P = .016), although a median OS could not be reliably estimated. In addition, patients who received midostaurin plus chemotherapy had a significant improvement in event-free survival compared with the placebo group, 8.2 months versus 3 months, respectively (HR, 0.78; 2-sided P = .005).

The most common (≥20%) adverse reactions in patients with AML who received midostaurin included febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infection.

The most common (≥20%) adverse reactions in patients with aggressive systemic mastocytosis or mast-cell leukemia included nausea, vomiting, diarrhea, edema, musculoskeletal pain, abdominal pain, fatigue, upper respiratory tract infection, constipation, fever, headache, and shortness of breath.

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