FDA approves Vyxeos for secondary acute myeloid leukemia in children
The FDA expanded the approval of daunorubicin and cytarabine to include the treatment of children aged at least 1 year with newly diagnosed therapy-related acute myeloid leukemia, according to a manufacturer-issued press release.
This indication for daunorubicin and cytarabine (Vyxeos, Jazz Pharmaceuticals) — a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor — also applies to children with AML with myelodysplasia-related changes.
The FDA based this approval, in part, on safety and pharmacokinetic data from two single-arm trials. In both the phase 1/phase 2 Children’s Oncology Group (COG) AAML1421 trial — which included 38 patients aged 1 to 21 years with AML in first relapse — and the phase 1 CPX-MA-1201 trial, which was conducted by Cincinnati Children's Hospital and included 27 patients aged 1 to 19 years with relapsed/refractory hematologic malignancies, researchers observed no safety differences based on patient age. Data on efficacy of the treatment were derived from an adult population of the phase 3 CPX-351 301 trial.
Common adverse events associated 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.
“The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients,” 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 COG myeloid disease committee, said in the release. “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.”