FDA approval of additional indication for Vyxeos for the treatment of secondary acute myeloid leukemia in pediatric patients.- Jazz Pharma

Jazz Pharmaceuticals plc has announced that the FDA approved a revised label for Vyxeos (daunorubicin and cytarabine) to include a new indication to treat newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged one year and older. The approval of Vyxeos for this indication is supported by safety data from two single-arm trials: AAML1421, conducted by the Children's Oncology Group (COG) and CPX-MA-1201, conducted by Cincinnati Children's Hospital (CCH) and evidence of effectiveness from an adequate and well-controlled study in adults. Safety and pharmacokinetics of Vyxeos in children and young adults were established in two clinical studies that enrolled patients with AML or relapsed/refractory hematologic malignancies. Thirty-eight pediatric patients aged one to 21 years of age with AML in first relapse were enrolled in the Phase 1/II AAML1421 study conducted by COG, and 27 patients aged one to 19 years with relapsed/refractory hematologic malignancies were enrolled in the Phase 1 CPX-MA-1201 study conducted by CCH. Both studies found no differences in the safety profile based on age. The use of Vyxeos for this indication is supported by evidence of effectiveness from study CPX351-301 in adult patients. Vyxeos has a Boxed Warning as it cannot be substituted with other daunorubicin and/or cytarabine-containing products. In the Phase III study, the most common adverse reactions (incidence greater than 25%) 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.