General AML

The FDA to review Vyxeos New Drug Application for AML

On 3rd 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.

References
  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. http://www.prnewswire.com/news-releases/jazz-pharmaceuticals-completes-rolling-submission-of-new-drug-application-for-vyxeos-cpx-351-an-investigational-treatment-for-acute-myeloid-leukemia-300433177.html [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.