Miltefosine (Oral)

Miltefosine is a drug used to treat leishmaniasis that manifests on the skin, in the internal organs and on the mucous membranes.


For many years, doctors around the world used antimony injections to treat the parasitic disease called leishmaniasis. Over the last several years, this treatment has proven to be ineffective for most types of leishmaniasis. Miltefosine is a newer treatment in an oral form that has proven to be up to 94 percent effective in eradicated leishmaniasis from the human body. It is indicated for the treatment of cutaneous leishmaniasis, mucosal leishmaniasis and visceral leishmaniasis. While the drug is approved by the United States Food and Drug Administration for treating these conditions, the drug is also effective in treating some rare conditions, and it used at times in the treatment of schistosomiasis, acanthamoeba keratitis, granulomatus amebic encephalitis, disseminated amebiasis and primary amebic encephalitis. Miltefosine was originally used as an anticancer drug, but it is no longer used for that purpose.

Leishmaniasis is a parasitic infection transmitted by the bite of a sand fly. The disease manifests itself as sores on the skin, in the mucous membranes and in the internal organs. Visceral leishmaniasis is fatal if not treated. As many 350 million people are affected by leishmaniasis around the world each year.

Miltefosine treats leishmaniasis in two ways. Miltefosine disturbs the mitochondrial function of the cells leading to cell death. It also disrupts protein kinase B within the parasite leading to cell death. There may be other ways in which the medication works, and doctors are still studying the exact mechanisms by which the medication cures leishmaniasis.

Miltefosine has some serious side effects associated with it. It must be kept in mind that this is the only oral drug available that cures leishmaniasis. Unless side effects are of a severe nature, patients will remain on the drug so that they will be cured of leishmaniasis which is potentially fatal.

Many of the most reported side effects in those taking the drug are associated with the gastrointestinal system. There are also many reports of elevated blood value counts associated with the function of the liver and the kidneys. Doctors will very closely monitor patients who are taking this medication. Frequent liver and kidney function blood test will be performed to monitor for potential damage to these organs. Very serious skin reactions have been reported in those using this medication. The Stevens-Johnson Syndrome, an extremely severe skin condition, has been reported with the use of Miltefosine.

This drug is known to cause birth defects in unborn children. Before a woman of childbearing years is placed on this medication, a pregnancy test should be performed. Those who are taking this drug should be on birth control during the time the drug is being taken and for a period of at least five months after the medication course is completed.

This medication is not approved for use in children under the age of twelve. The drug should not be used in anyone weighing less than 66 pounds.

Conditions Treated

  • Visceral leishmaniasis

Indicated with orphan drug status for the following:

  • Schistosomiasis

Type Of Medication

  • Anti-leishmaniasis medication

Side Effects

Miltefosine has several types of side effects that have been reported in over ten percent of the patients using the drug. The most widely reported side effects are associated with the rise in serum blood levels in the patient. For instance, 94 percent of patients had an increase in serum transaminases. 62 percent of patients had a decreased platelet count while on the drug. 25 percent of patients had an increase in serum creatinine levels indicated possible kidney malfunction. Other hematological side effects have been reported including thrombocytopenia and agranulocytosis.

The next most significant group of side effects occurred in the gastrointestinal system. Nausea occurs in over 40 percent of those on Miltefosine. Almost 40 percent of those on the drug report vomiting, and approximately 20 percent of patients report diarrhea. Over 11 percent of patients reported pain in the abdomen while about 10 percent reported a decrease in appetite.

Neurological side effects were the next largest group reported. Almost 30 percent of patients reported headache and nearly the same number of patients reported motion sickness.

Skin side effects must be carefully monitored while on miltefosine. While it is a rare occurrence, there have been reports of the Stevens-Johnson Syndrome in those using miltefosine. Itching of the skin and skin rash are not uncommon side effects.

In addition to these more common and serious side effects, the following side effects have also been reported in those using miltefosine:

  • Asthenia


This medication is supplied in 50 milligram capsules for oral use. The dosage of miltefosine is weight dependent. Patients who weigh more than 45 kilograms should receive one capsule three times per day for a period of 28 days. Those who weigh under 45 kilograms should receive one capsule two times per day for a period of 28 days.

Patients who weigh over 165 pounds will receive an individualized dose as determined by the healthcare provider.

It is important to take the drug for the full 28 days. Failure to do so may lead the parasites not being completely destroyed within the body.


It is important that patients inform their healthcare professional regarding all prescription and nonprescription drugs that they are currently taking. The following list of drugs contains drugs that may interact with miltefosine. Use of these drugs will not necessarily cause the patient not to be able to use miltefosine. Doctors will need to monitor patients for possible reactions if they are taking the following:

  • Acemetacin


Those patients who have liver or kidney disease should notify their healthcare professional before taking this medication. This drug causes increases in the blood levels of liver and kidney enzymes. The doctor will perform liver and kidney function blood tests while the patient is on this drug.

Pregnant women should not use miltefosine. The doctor will perform a pregnancy test on all women of childbearing years before the drug is prescribed. This medication in listed as Pregnancy Category D. It is known to cause severe birth defects and fetal harm to the unborn child. Women should be on birth control during the time that they are taking miltefosine and for a period of at least five months after the last dose of miltefosine is taken.

Women who are breastfeeding should not take miltefosine. It is not known if this drug is transmitted through the breast milk.

Miltefosine is not approved for those under the age of 12. No one who is under 66 pounds should take the drug. Those who weigh more than 165 pounds will need to receive an adjusted dose of the drug determined by the prescribing doctor.

Miltefosine is know to cause severe skin reactions. Skin blistering and peeling have been reported. The drug is known to cause the Stevens-Johnson Syndrome which is an extremely severe skin condition. If a patient notices that they are having generalized skin redness or a rash, the patient should notify the physician immediately.

It is important that patients finish the entire course of this medication. Failure to finish the complete course may result in the parasites not being completely eradicated from the body. If not completely eradicated, they will once again proliferate and cause serious harm to the patient.

Patients with Sjogren -Larsson Syndrome should not receive miltefosine.


Miltefosine should be stored in a dry place with a temperature between 68 and 77 degrees Fahrenheit.


Miltefosine is marketed under the brand name of Impavido. It is the only oral drug that is known to treat leishmaniasis. The drug treats the three major forms of the disease including mucosal, visceral and cutaneous leishmaniasis. Leishmaniasis is a very serious condition. Visceral leishmaniasis if fatal if not treated. Miltefosine can be used to treat some rare amebic conditions for which no other good drug treatments exist.

There are some serious side effects associated with the use of this medication. The most noted side effects are elevations of liver enzymes and creatinine in the blood. Elevation of these levels indicate possible liver and kidney malfunction. Doctors will perform liver and kidney function tests on the patient during the course of therapy to make sure that damage to the liver and the kidneys is not occurring.

Gastrointestinal side effects are also very common. Patients reported nausea, vomiting and diarrhea at high levels. Over 30 percent of patients will experience some type of gastrointestinal symptom while on the medication.

Miltefosine is a Pregnancy Category D drug. It causes fetal harm. Pregnant women should not use this drug. Women of childbearing years should use birth control while on the medication and for a period of at least five months after the medication is ended.

The proper dosage of miltefosine is dependent on the patient's weight. Those under twelve and those under 66 pounds should not use this medication. Those over 165 pounds will have a specific dose determined by the prescribing physician.

Patients with Sjogren-Larsson Syndrome should not receive miltefosine.