CPX-351 granted approval in the EU for the treatment of therapy-related acute myeloid leukemia or AML-MRC

On 27^th August 2018, the European Commission (EC) granted approval to CPX-351,  44 mg/100 mg powder for concentrate for solution for infusion for the treatment for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). This comes after the positive opinion recommending a marketing authorization for CPX-351 was given by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP).¹

The EC approval for CPX-351, a liposomal formulation of daunorubicin plus cytarabine co-encapsulated at a molar ratio of 1:5, was based on clinical data from five studies including the pivotal phase III study (NCT01696084), which evaluated the safety and efficacy of Vyxeos^® compared to cytarabine and daunorubicin (7+3) in patients with newly diagnosed t-AML and AML-MRC. The results of this pivotal study were recently published by Jeffery Lancet et al. in the Journal of Clinical Oncology.²

Three-hundred and nine patients with newly diagnosed sAML were randomly assigned 1:1 to receive Vyxeos^® (n = 153, mean age = 67.8 years) or 7+3 (n = 156, mean age = 67.7) as induction and consolidation chemotherapy. The primary endpoint of the study, overall survival (OS), was met with patients in the CPX-351 arm significantly had a longer OS compared to patients in the 7+3 arm (Median OS in the CPX-351 and 7+3 arms: 9.56 vs 5.95 months respectively, HR = 0.69 [95% CI, 0.52–0.90], P = 0.003).²

According to the drug manufacturer’s, Jazz Pharmaceuticals, “CPX-351is the first chemotherapy to demonstrate an OS advantage versus the standard of care in a phase III study of older adult patients with newly diagnosed t-AML or AML-MRC”.¹