Drug Evaluation

Department of Leukemia, University of Texas, MD Anderson Cancer Center, Houston, TX, USA

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Varsha V Gandhi

Department of Experimental Therapeutics, University of Texas, MD Anderson Cancer Center, Houston, TX, USA

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Farhad Ravandi

* Author for correspondence

Department of Leukemia, University of Texas, MD Anderson Cancer Center, Houston, TX, USA. .

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Email the corresponding author at fravandi@mdanderson.org

Published Online:31 Jul 2013https://doi.org/10.2217/fon.13.130

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Abstract

Elacytarabine (formerly CP-4055) is a lipid-conjugated derivative of the nucleoside analog cytarabine. Elacytarabine was rationally designed to circumvent cytarabine resistance related to decreased cellular uptake, due to the ability of the lipophilic drug moiety to enter the cell without the requirement of specialized nuclear transport proteins, including the hENT1. In preclinical and clinical studies, elacytarabine has demonstrated both safety and efficacy in acute myeloid leukemia (AML), with noteworthy activity among the cytarabine-refractory AML population. Elacytarabine was granted orphan drug designation status from the European Commission in 2007 and from the US FDA in 2008, with a fast-track approval designation from the FDA in 2010. Results of a recent randomized Phase III clinical trial, however, failed to show superiority of elacytarabine over the investigator’s choice of therapy for relapsed or refractory AML.

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Published online 31 July 2013

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Financial & competing interests disclosure

F Ravandi has been a member of advisory boards for Clavis Pharmaceutics and has received honoraria. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

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Elacytarabine (CP-4055) in the treatment of acute myeloid leukemia

Abstract

References