What is the role of miltefosine in the treatment of leishmaniasis?

Updated: Feb 18, 2020
  • Author: Craig G Stark, MD, FACP, FFTM, RCPS(Glasg), FISTM; Chief Editor: Pranatharthi Haran Chandrasekar, MBBS, MD  more...
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Miltefosine is the sole oral agent that has been shown to be effective against leishmaniasis. This medication was developed first as an antineoplastic agent and later found to have considerable antiproliferative activity against leishmaniasis as well as against other trypanosome parasites. It is an attractive agent in areas, such as India, that have drug resistance against traditional chemotherapy.

In August 2013, the CDC made available an expanded access investigational new drug (IND) protocol for miltefosine for treatment of free-living amebae (FLA) in the United States. [40] In March 2014, the CDC approved miltefosine for the treatment of specific species that cutaneous, mucosal, and visceral leishmaniasis, in adults and adolescents who aged at least 12 years, weigh at least 66 lb, and are not pregnant or breastfeeding. [2]

This medication is approved in India for visceral leishmaniasis.

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