At the end of March 2021, the Food and Drug Administration (FDA) approved expanded use for Vyxeos (daunorubicin and cytarabine) that now includes the ability to treat newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged 1 and older. 

Vyxeos is a liposomal combination of daunorubicin (an anthracycline topoisomerase inhibitor) and cytarabine (a nucleoside metabolic inhibitor). This drug was approved for pediatric use based on safety data from two single-arm trials, AAML1421 and CPX-MA-1201, as well as efficacy data from the CPX 351-301 study in adults.

Dr. Edward Anders Kolb, who is the director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children and chair of the Myeloid Disease Committee at Children's Oncology Group (COG) conducted the AAML1421 trial. In a press release, he stated that “The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients." 

Vyxeos was evaluated in children and young adults with AML or relapsed/refractory blood cancers. Thirty eight pediatric patients aged 1 to 21 years with secondary AML participated in the phase 1/2 AAML1421 study and 27 patients ages 1 to 19 years with relapsed/refractory blood cancers participated in the phase 1 CPX-MA-1201 study. The studies revealed that there was no difference in the safety profile from either study based on age.

According to the phase 3 CPX351-301 study, the most common side effects 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.

Vyxeos has been used in adult patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes since 2017.