FDA Approval Summary: (Daunorubicin and Cytarabine) Liposome for Injection for the Treatment of Adults with High-Risk Acute Myeloid Leukemia

On August 3, 2017, the Food and Drug Administration (FDA) granted regular approval to Vyxeos (also known as CPX-351), a liposomal formulation of daunorubicin and cytarabine in a fixed combination, for the treatment of adults with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Approval was based on data from Study CLTR0310-301, a randomized, multicenter, open-label, active-controlled trial comparing Vyxeos to a standard combination of daunorubicin and cytarabine ("7+3") in 309 patients 60-75 years of age with newly-diagnosed t-AML or AML-MRC. Due to elemental copper concerns with the Vyxeos formulation, patients with Wilson's disease were excluded from the study. Vyxeos demonstrated an improvement in overall survival (HR 0.69; 95% CI: 0.52, 0.90; p=0.005) with an estimated median overall survival (OS) of 9.6 months compared with 5.9 months for the "7+3" control arm. The toxicity profile of Vyxeos was similar to that seen with standard "7+3" with the exception of more prolonged neutropenia and thrombocytopenia on the Vyxeos arm. Because the pharmacology of Vyxeos differs from that of other formulations of daunorubicin and cytarabine, labeling includes a warning against interchanging formulations during treatment. This is the first FDA-approved treatment specifically for patients with t-AML or AML-MRC.

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