Miltefosine: Uses, Contraindications, Side Effects, and Mechanism of Action

Initially developed as an anti-cancer drug, Miltefosine is chemically a phosphorylcholine ester of hexadecanol. It is an anti-leishmanial drug. Miltefosine is the first medicine to be given orally as a membrane-active alkyl phospholipid to treat “Cutaneous and Visceral leishmaniasis”. It is an oral medicine that, unlike earlier traditional therapy such as pentavalent antimonial drugs, amphotericin B, and pentamidine, which are parenteral agents, increases the treatment base in the population and lowers healthcare expenses. Miltefosine is more effective than antifungal amphotericin B and other conventional therapies against resistant antimonial Leishmania isolates and has fewer adverse effects.

Miltefosine Mechanism of Action

The exact mechanism of action of the drug Miltefosine is unknown. However, the proposed mechanism of action of anti-leishmanial activity of the drug Miltefosine suggests that inside the infected-Leishmania cell the drug shows various effects via its detergent-like action disrupting intracellular membranes of the cell, ultimately resulting in a mechanism similar to apoptosis.

Miltefosine Pharmacokinetics

The drug is well absorbed following oral administration. The Bio-half-life of the drug is 150 to 200 hours after oral administration and clearance are 25-30 days. The drug shows minimal urinary excretion. Intact hepatocytes have a low choline release, with decreasing rates in the provided order:


What is miltefosine used for?

  • Cutaneous leishmaniasis
  • Visceral leishmaniasis (more severe form, lethal if the disease is untreated)
  • HIV patients with visceral leishmaniasis

According to a study published in 2004, Leishmaniases were found in 88 countries, with a mortality of 12–14 million persons and 1.5–2 million new cases each year. The global death rate was around 60,000 persons per year among the 350 million people who live in endemic areas.

0.40.5 million cases of Visceral leishmaniasis occur in

  • India
  • Nepal
  • Bangladesh
  • Brazil
  • Sudan

1-1.5 million cases of Cutaneous leishmaniasis occur in

  • Afghanistan
  • Brazil
  • Iran
  • Sudan
  • Peru

Current stats show approximately 50,000 cases of Visceral leishmaniasis and around 1.5 M cases of Cutaneous Leishmaniasis occur annually with more than ninety percent cases of visceral leishmaniasis occurring in

  • India
  • Nepal
  • Bangladesh
  • Sudan
  • Ethiopia
  • Brazil

Out of all cases of cutaneous leishmaniasis, approximately 90% occur in

  • Brazil
  • Peru
  • Algeria
  • Saudi Arabia
  • Syria
  • Iraq
  • Iran
  • Afghanistan

Miltefosine Contraindications

1.      Pregnancy and Child-bearing age

Miltefosine is a toxin that causes birth defects (teratogenic). It should not be used during pregnancy. Because the medicine has a long half-life, contraception should be provided for at least two months if a woman of childbearing age is receiving treatment.

Is Meltifosine safe in pregnancy?

FDA assigns category D to this drug. Miltefosine should not be used if you are pregnant. Before beginning this medication, you may need to obtain a negative pregnancy test.

2.      Sjogren Larsson Syndrome

Patients with Sjogren Larsson Syndrome are unable to metabolize long-chain fatty alcohols like hexadecanol, and the buildup of this hexadecanol causes severe neurological abnormalities beginning in childhood. Because hexadecanol, long-chain fatty alcohol derived from Miltefosine disintegration, can be converted to palmitic acid and used in lipid biosynthesis or beta-oxidation, the therapy is not recommended for people who have a genetic abnormality connected to the rare Sjogren Larsson syndrome.

Miltefosine Side effects

  • Gastrointestinal toxicity (Mild to Moderate)
  • Nausea
  • Elevated levels of serum creatinine
  • Vomiting
  • Elevated serum aspartate aminotransferase
  • Elevated serum alanine aminotransferase

Is Miltefosine Available in India?

India accounts for more than half of the world’s kala-azar burden. In 2005, Miltefosine was commonly available without a prescription or a limit on the amount dispensed over the counter (OTC). When the medicine was marketed in private pharmacies in India, the possibility for resistance became a serious worry, and patients were compelled to accept shorter courses due to financial constraints. As a result, from 2008 forward, India only provided miltefosine to the public sector. Miltefosine’s effectiveness is said to have dropped after being taken as monotherapy for over a decade.

Miltefosine Brand names

  • Miltex
  • Impavido
  • Miteforan


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