Miltefosine (Impavido): the first oral treatment against leishmaniasis

H Sindermann1, S L Croft, K R Engel

  • 1Zentaris AG, Weismüllerstrasse 45, 60314 Frankfurt am Main, Germany.

Insights

Miltefosine offers an effective oral treatment for visceral leishmaniasis, showing high cure rates even in patients resistant to antimonial drugs. This novel antileishmanial drug provides a significant advantage over injectable therapies.

Area of Science:

  • Pharmacology
  • Infectious Diseases
  • Drug Discovery

Background:

  • Leishmaniasis remains a significant global health challenge, particularly visceral leishmaniasis.
  • Current treatments for leishmaniasis often require parenteral administration and face challenges with drug resistance.
  • There is a need for novel, orally available antileishmanial agents.

Purpose of the Study:

  • To evaluate the clinical efficacy of miltefosine as an oral treatment for visceral leishmaniasis.
  • To assess the effectiveness of miltefosine in patients resistant to standard antimonial therapies.
  • To highlight the advantages of oral miltefosine over parenteral treatments.

Main Methods:

  • In vitro and in vivo studies were conducted to assess drug selectivity.
  • Clinical trials were performed to demonstrate the efficacy of oral miltefosine.
  • Patient outcomes were compared, including those with resistance to existing therapies.

Main Results:

  • Miltefosine demonstrated significant selectivity in preclinical models.
  • Clinical studies confirmed the efficacy of miltefosine for visceral leishmaniasis treatment.
  • High cure rates were achieved with oral miltefosine, including in patients refractory to antimonial drugs.

Conclusions:

  • Miltefosine is a novel, orally administered drug effective against visceral leishmaniasis.
  • Its oral bioavailability and efficacy against resistant strains offer a significant therapeutic advantage.
  • Miltefosine represents a promising alternative to parenteral treatments for leishmaniasis.

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