FDA Clears Miltefosine (Impavido) for Leishmaniasis

Megan Brooks

March 20, 2014

The US Food and Drug Administration (FDA) on March 19 approved oral miltefosine (Impavido, Paladin Therapeutics) to treat visceral, cutaneous, and mucosal leishmaniasis in patients 12 years of age and older.

Miltefosine is the first FDA-approved drug to treat cutaneous or mucosal leishmaniasis.

The FDA's Anti-Infective Drugs Advisory Committee endorsed miltefosine for leishmaniasis in October 2013, as reported by Medscape Medical News.

Leishmaniasis is caused by the Leishmania parasite, which is transmitted by the bite of sand flies. The disease occurs primarily in people who live in the tropics and subtropics. Miltefosine has been used for leishmaniasis in countries where the parasitic infection is endemic.

Leishmaniasis is rare in developed countries, typically only being imported by travelers and members of the military. It is classified as a neglected tropical disease.

The approval of miltefosine "demonstrates the FDA's commitment to making available therapeutic options to treat tropical diseases," Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research, said in a statement.

Miltefosine had FDA fast-track designation, priority review, and orphan product designation because the drug "demonstrated the potential to fill an unmet medical need in a serious disease or condition [and] the potential to be a significant improvement in safety or effectiveness in the treatment of a serious disease or condition and is intended to treat a rare disease," the FDA said.

Boxed Warning

Miltefosine was studied in 4 clinical trials, in which a total of 730 patients received miltefosine or a comparator drug or placebo. In testing, miltefosine proved "safe and effective in treating visceral, cutaneous and mucosal leishmaniasis," the FDA said.

The most common adverse effects seen in clinical trials were nausea, vomiting, diarrhea, headache, decreased appetite, dizziness, abdominal pain, itching, drowsiness, and elevated levels of liver enzymes and creatinine.

The labeling for the drug includes a boxed warning to alert patients and healthcare providers that the drug can cause fetal harm and should not be given to pregnant women. Healthcare professionals should advise women to use effective contraception during and for 5 months after treatment with miltefosine, the FDA said.


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