CPX-351 (vyxeos) in AML

Abstract

For decades, the standard induction for patients with acute myeloid leukemia (AML) has been the combination of cytarabine with anthracycline (7 + 3 regimen). In August 2017 the US FDA approved CPX-351 (vyxeos), a liposomal formulation of cytarabine and daunorubicin at a fixed 5:1 molar ratio, for the treatment of adults with newly diagnosed AML with myelodysplasia-related changes (AML-MRC) and therapy-related AML (t-AML). This is the first approved treatment specifically for patients with this subgroup of AML. The approval was based on findings from a multicenter, randomized, open-label, phase III study of CPX-351 Versus 7 + 3 in patients 60–75 years old with newly diagnosed AML-MRC or t-AML. In this study CPX-351 had a higher median OS than 7 + 3 (9.56 vs 5.95 months, HR 0.69; 95% CI: 0.52 to 0.90, p = 0.005). In this profile, we review preclinical and clinical data, and discuss limitations and future directions with CPX-351 use in AML.

Keywords:

• CPX-351
• Vyxeos
• t-AML
• sAML
• AML-MRC
• AML in elderly

Disclosure statement

Hagop Kantarjian, Tapan Kadia, Farhad Ravandi-Kashani and Naval Daver received research funding and consultancy/advisory from jazz pharmaceuticals.

Additional information

Funding

This work was supported in part by the MD Anderson Cancer Centre Support Grant (CCSG) CA016672, The MD Anderson Cancer Center Leukemia SPORE CA100632, the Charif Souki Cancer Research Fund and generous philanthropic contributions to the MD Anderson Moon Shots Program.