Revised Label Approved by FDA for Daunorubicin/Cytarabine Combination for AML
Posted: Friday, April 2, 2021
On March 30, the U.S. Food and Drug Administration (FDA) approved an updated label for the daunorubicin and cytarabine (Vyxeos) combination therapy to include a new indication for therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in pediatric patients 1 year and older. The therapy is a liposomal combination of an anthracycline topoisomerase inhibitor and a nucleoside metabolic inhibitor. For more prescribing information visit accessdata.fda.gov.
The FDA approval for the new indication is based on safety and efficacy data from the phase 1/2 AAML1421 trial and the phase I CPX-MA-1201 clinical trial. Both studies enrolled patients with AML or relapsed/refractory hematologic malignancies. About 38 pediatric patients who were 21 years or older enrolled in the AAML1421 study, and 27 patients who were 1 to 19 years were enrolled in the CPX-MA-1201 study.
Clinical trial results from both studies found no differences in safety profile based on age. The indication is supported by evidence of effectiveness from the CPX351-301 trial in adult patients.