The U.S. Food and Drug Administration approved CPX-351, a fixed combination of cytarabine and daunorubicin, for the treatment of adults with newly diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Both are considered high-risk AML subtypes.
The drug was approved based on results from a study of 309 patients with newly diagnosed t-AML or AML-MRC who were randomized to receive daunorubicin and cytarabine either in the fixed-combination formulation or as separate treatments. Patients who received CPX-351 had a longer median overall survival compared with those who received separate treatments (9.56 months vs. 5.95 months).
Common adverse events associated with CPX-351 included hemorrhage, febrile neutropenia, rash, edema, nausea, mucositis, diarrhea, constipation, musculoskeletal pain, and fatigue.
Patients who have a history of serious hypersensitivity to daunorubicin, cytarabine, or any component of the formulation should not use CPX-351, and the prescribing information includes a boxed warning not to interchange CPX-351 with other daunorubicin- and/or cytarabine-containing products.
Source: FDA press release, August 3, 2017.