VyxeosNewly-diagnosed therapy-related acute myeloid leukaemia (AML) in children aged one year and older
Development and Regulatory status
Mar 21Approved in US for the treatment of newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes (AML-MRC) in paediatric patients 
Jan 12Granted orphan drug status in EU (EU/3/11/942) .
Two DNA synthesis inhibitors - cytarabine inhibits pyrimidine synthesis & daunorubicin inhibits DxNA topoisomerase I. Cytarabine with daunorubicin is encapsulated in the liposome in a 5:1 molar ratio for maximum synergistic benefit.
Around 70 of new cases of childhood AML are diagnosed every year in the UK, which accounts for 15% of children’s leukaemia cancer cases. Incidence varies with age. The highest risk is in children aged less than two years; the risk in children aged two to nine years is lower and it then rises through the adolescent years .
Newly-diagnosed therapy-related acute myeloid leukaemia (AML) in children aged one year and older
Trial or other data
Dec 19Results posted for PI/II study (NCT02642965). 68.30% (52.89 to 78.02) of patients were considered responders after up to 2 cycles .
Apr 16PI/II trial to determine a recommended P2 dose (RP2D) and the toxicities associated with liposome-encapsulated daunorubicin-cytarabine (CPX-351) in pediatric and young adult patients with relapsed/refractory acute myeloid leukemia (AML), and to estimate the response rate (complete remission [CR] plus complete remission with partial platelet recovery [CRp]) after CPX-351 (cycle 1) followed by fludarabine phosphate, cytarabine, and filgrastim (FLAG) (cycle 2) in children with AML in first relapse starts (NCT02642965). 38 children and young adults aged up to 21 years will be recruited in the US & Canada. Collection of primary outcome data is due to complete Dec 18