The Food and Drug Administration (FDA) has expanded the approval of Vyxeos® (daunorubicin and cytarabine; Jazz Pharmaceuticals) to include the treatment of patients aged 1 year and older with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Previously, Vyxeos was only approved for adults.

The approval was based on data from 2 single-arm trials, AAML1421 ( NCT02642965) and CPX-MA-1201 ( NCT01943682), that evaluated the safety and pharmacokinetics of Vyxeos in patients aged 1 year and older. The phase 1/2 AAML1421 study included 38 patients aged 1 to 21 years with AML in first relapse, and the phase 1 CPX-MA-1201 study included 27 patients aged 1 to 19 years with relapsed/refractory hematologic malignancies. 

Findings from both studies showed there were no differences in the safety profile of Vyxeos based on age. Moreover, the efficacy of Vyxeos for this indication is supported by previously reported data from the phase 3 CPX351-301 study ( NCT01696084) in adult patients.

Vyxeos carries a Boxed Warning stating it is not interchangeable with other daunorubicin- or cytarabine-containing products. The most common adverse reactions (incidence of greater than or equal to 25%) reported in the phase 3 study were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders and vomiting.

“The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients,” said Dr. Edward Anders Kolb, MD, director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children and chair of myeloid disease committee at COG. “Jazz has been a wonderful partner in pediatric drug development and we are grateful for the continued work being done to provide safe and effective therapies for children.”

Vyxeos is supplied as a lyophilized cake containing 44mg of daunorubicin and 100mg of cytarabine in single-dose vials in 2- and 5-count cartons.


1.    Jazz Pharmaceuticals announces FDA approval of additional indication for Vyxeos® (daunorubicin and cytarabine) for the treatment of secondary acute myeloid leukemia in pediatric patients. [press release]. Dublin, Ireland: Jazz Pharmaceuticals plc; March 30, 2021. 

2.    Vyxeos [package insert]. Palo Alto, CA: Jazz Pharmaceuticals, Inc.; 2021.