Daunorubicin and Cytarabine Combination Approved for Pediatric Secondary AML

The U.S. Food and Drug Administration (FDA) has approved a revised label for liposomal daunorubicin and cytarabine, CPX 351, to include a new indication for the treatment of newly diagnosed secondary acute myeloid leukemia (AML) or AML with myelodysplasia-related changes in children aged one year and older.

The decision to extend the approval to younger patients is supported by safety data from the phase I/II AAML1421 trial, which enrolled 38 patients with AML aged one to 21 years, and the phase I CPX-MA-1201 study, which included 27 patients aged one to 19 years with relapsed or refractory hematologic malignancies. Investigators found no difference in safety based on age.

“The expansion of the label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients,” said Edward Anders Kolb, MD, director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. duPont Hospital for Children.

Source: Jazz Pharmaceuticals press release, March 30, 2021.