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Miltefosine

Miltefosine
Miltefosine structure.svg
Clinical data
Trade names Impavido, Miltex, others
AHFS/Drugs.com Monograph
Pregnancy
category
  • US: D (Evidence of risk)
Routes of
administration
By mouth
ATC code
Legal status
Legal status
  • US: ℞-only
  • In general: ℞ (Prescription only)
Pharmacokinetic data
Bioavailability High
Protein binding ~98%
Metabolism Slow hepatic (non--dependent)
Biological half-life 6 to 8 days and 31 days
Excretion Primarily fecal
Identifiers
CAS Number
PubChem CID
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
NIAID ChemDB
ECHA InfoCard 100.151.328
Chemical and physical data
Formula C21H46NO4P
Molar mass 407.568 g/mol
3D model (Jmol)
 NYesY (what is this?)  

Miltefosine, sold under the trade name Impavido among others, is a medication mainly used to treat leishmaniasis and free-living amoeba infections such as Naegleria fowleri. This includes leishmaniasis of the cutaneous, visceral, and mucosal types. It may be used together with liposomal amphotericin B or paromomycin. It is taken by mouth.

Common side effects include vomiting, abdominal pain, fever, headaches, and decreased kidney function. More severe side effects may include Stevens-Johnson syndrome or low blood platelets. Use during pregnancy appears to cause harm to the baby and use during breastfeeding is not recommended. How it works is not entirely clear.

Miltefosine was first made in the early 1980s and studied as a treatment for cancer. A few years later it was found to be useful for leishmaniasis and was approved for this use in 2002 in India. It is on the World Health Organization's List of Essential Medicines, the most effective and safe medicines needed in a health system. In the developing world a course of treatment costs 65 to 150 USD. In the developed world treatment may be 10 to 50 times greater.

Miltefosine is primarily used for the treatment of visceral and New World cutaneous leishmaniasis, and is undergoing clinical trials for this use in several countries, such as Brazil,Guatemala. and the United States. This drug is now listed as a core medication for the treatment of leishmaniasis under the WHO Model List of Essential Medicines. Several medical agents have some efficacy against visceral or cutaneous leishmaniasis, however, a 2005 survey concluded that miltefosine is the only effective oral treatment for both forms of leishmaniasis. In addition, it has been used successfully in some cases of the very rare, but highly lethal, brain infection by the amoeba, Naegleria fowleri, acquired through water entering the nose during a plunge in contaminated water.


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