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The FDA to review Vyxeos New Drug Application for AML

Apr 6, 2017

On 3 rdApril 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 presentedat the American Society of Clinical Oncologymeeting 2016 by Jeffrey E. Lancetfrom 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 vs5.95 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% vs33.3%, 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.

  1. PRNewswire: Jazz Pharmaceuticals Completes Rolling Submission of New Drug Application for Vyxeos™ (CPX-351), an Investigational Treatment for Acute Myeloid Leukemia. 2017 Apr 3.[Accessed 2017 Apr 6].
  2. Lancet J.E. et al. Final results of a phase III randomized trial of CPX-351 versus 7+3 in older patients with newly diagnosed high risk (secondary) AML. Oral Abstract #7000. American Society of Clinical Oncology Meeting 2016.