FDA Approves First New Drug for AML Since 1990

Nick Mulcahy

April 28, 2017

The US Food and Drug Administration (FDA) today approved the kinase inhibitor midostaurin (Rydapt, Novartis), in combination with chemotherapy, for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who have the FLT3 genetic mutation.

Midostaurin is the first new drug approved for the treatment of AML since 1990.

The drug is approved for use with a companion diagnostic, the LeukoStrat CDx FLT3 Mutation Assay (Invivoscribe Technologies). The mutation is seen in 30% to 35% of patients with AML and is associated with a worse prognosis.

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

The safety and efficacy of midostaurin for treatment-naive patients with AML were studied in a randomized trial of 717 patients who had not been treated previously for AML, as reported by Medscape Medical News in 2015, when phase 3 clinical trial results were first reported at the annual meeting of the American Hematology Society.

At the time, a number of experts called midostaurin the new standard of care for these patients.

In the trial, patients who received midostaurin in combination with chemotherapy lived longer than patients who received chemotherapy alone. However, the FDA observed that a specific median survival rate "could not be reliably estimated."

In addition, the median event-free survival rate in the midostaurin-chemotherapy group was 8.2 months vs 3 months for the chemotherapy-alone group.

The most common adverse events with midostaurin include febrile neutropenia, nausea, mucositis, vomiting, headache, petechiae, musculoskeletal pain, epistaxis, device-related infection, hyperglycemia, and upper respiratory tract infection.

Women who are pregnant or breastfeeding should not take midostaurin because it may cause harm to a developing fetus or a newborn baby. Patients who experience signs or symptoms of pulmonary toxicity should stop using midostaurin.

Midostaurin was also approved today for adults with the rare blood disorders aggressive systemic mastocytosis and systemic mastocytosis with associated hematologic neoplasm or mast cell leukemia. Common side 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.

Follow Medscape senior journalist Nick Mulcahy on Twitter: @MulcahyNick

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