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FDA Approves Rx/Dx Combination for Acute Myeloid Leukemia

NEW YORK (GenomeWeb) – The US Food and Drug Administration today approved Novartis' acute myeloid leukemia drug Rydapt (midostaurin) in combination with chemotherapy alongside Invivoscribe Technologies' LeukoStrat CDx FLT3 Mutation Assay to identify patients who can receive the drug.

"Rydapt is the first targeted therapy to treat patients with AML, in combination with chemotherapy," Richard Pazdur, acting director of the Office of Hematology and Oncology Products in the FDA’s drug center, said in a statement. The FDA approved Rydapt on an accelerated timeline, and the AML indication received breakthrough therapy designation.

Adult AML patients may be eligible for Rydapt if the PCR-based LeukoStrat CDx detects an FLT3 mutation in their blood or bone marrow samples. "The ability to detect the gene mutation with a diagnostic test means doctors can identify specific patients who may benefit from this treatment," Pazdur added.

To approve the drug/test combination, the FDA reviewed data from a randomized study of more than 700 patients who hadn't previously been treated for AML. The study couldn't yield a specific median survival rate, but showed that patients receiving Rydapt and chemotherapy in combination lived longer than those receiving just chemotherapy. Patients on the combined regimen also did better on other measures such as failure to reach complete remission, disease progression, and death compared to those in the chemotherapy arm.