On March 30, 2021, the FDA approved a new indication for daunorubicin and cytarabine (Vyxeos; Jazz Pharmaceuticals) for the treatment of pediatric patients aged ≥1 years with newly diagnosed, therapy-related acute myeloid leukemia (AML) or patients with AML and myelodysplasia-related changes. Daunorubicin and cytarabine is a liposomal combination of an anthracycline topoisomerase inhibitor (daunorubicin) and a nucleoside metabolic inhibitor (cytarabine).
The FDA has previously approved the combination of daunorubicin and cytarabine for the treatment of adults with newly diagnosed, therapy-related AML or AML with myelodysplasia-related changes.
The FDA approved this new indication in pediatric patients based on safety data from 2 single-arm clinical trials: the AAML1421 study and the CPX-MA-1201 study, as well as on efficacy evidence shown in a previous clinical trial in adults.
“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 Edward Anders Kolb, MD, Director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children, and Chair of the Myeloid Disease Committee of the Children’s Oncology Group.
In all, 38 pediatric patients aged 1 year to 21 years with AML who were in first relapse were enrolled in the phase 1/2 AAML1421 study, and 27 patients aged 1 year to 19 years with relapsed or refractory hematologic malignancies were enrolled in the phase 1 CPX-MA-1201 study. In both studies, no differences were found in the safety profile of daunorubicin and cytarabine based on the patients’ age.
The most common (≥25%) adverse reactions with daunorubicin and cytarabine include 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.