The FDA to review Vyxeos New Drug Application for AML

On 3^rd April 2017, a New Drug Application (NDA) for vyxeos (CPX-351), a liposomal formulation of cytarabine plus daunorubicin co-encapsulated at a molar ratio of 5:1, was submitted to the U.S. Food and Drug Administration (FDA) for the treatment of patients with Acute Myeloid Leukemia (AML).¹

The NDA submission was based on clinical data from five studies including a pivotal phase III study (NCT01696084), which was presented at the American Society of Clinical Oncology meeting 2016 by Jeffrey E. Lancet from the Lee Moffitt Cancer Center & Research Institute, Florida, USA, and colleagues.²

In this phase III study, 309 newly diagnosed AML patients (60–75 years) were randomized to receive either CPX-351 (n = 153) or 7+3 (cytarabine and daunorubicin, n = 156) induction therapy. The endpoints of the study were Overall Survival (OS), Event Free Survival (EFS), and Complete Remission plus Complete Remission with Incomplete blood count recovery (CR+CRi).

The key results of the study were:

• Median OS in patients receiving CPX-351 and 7+3; 9.56 vs 5.95 months, HR = 0.69, P = 0.005
• EFS was superior in patients receiving CPX-351 compared to 7+3; HR = 0.74, P = 0.021
• CR+CRi response in patients receiving CPX-351 and 7+3; 47.7% vs 33.3%, P = 0.016

In summation, CPX-351 improved the survival in older patients with AML. Currently, CPX-351 is being investigated in a multiple study in newly diagnosed and relapsed/refractory AML patients.