By: Sang Hyo Kim, Staff Editor
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On March 19, 2014, the U.S Food and Drug Administration (FDA) approved miltefosine (Impavido®) for the treatment of leishmaniasis. Leishmaniasis is a disease caused by leishmania, a parasite that is transmitted to humans through sand fly bites. Although the majority of people affected with leishmaniasis are from tropical and subtropical regions, many U.S patients contract the disease when overseas.1 Leishmaniasis is found in over 80 countries, and about 350 million people are considered to be at risk. There are currently 12 million people infected and an estimated 70,000 deaths annually.2
Miltefosine, manufactured by Paladin Therapeutics, is the first oral drug approved to treat the three main types of leishmaniasis: visceral leishmaniasis (affecting internal organs), cutaneous leishmaniasis (affecting the skin), and mucosal leishmaniasis (affecting the nose and throat).1 Miltefosine has a direct toxic effect on the parasite by stimulating immune cells such as macrophages to cause parasite elimination via oxidative radicals.3
The efficacy of miltefosine was tested in four clinical trials with a total of 547 patients receiving the treatment drug and 183 patients receiving an alternative drug or placebo.1 The overall results showed that miltefosine can successfully treat the three main types of leishmaniasis. The FDA also granted miltefosine a fast track designation, priority review, and orphan designation. In other words, the drug demonstrated the potential to fill an unmet medical need in a serious disease state, the potential to be a significant improvement in a treatment of a disease, and the potential to treat a rare disease, respectively.4
Miltefosine is intended for patients 12 years of age and older.1 The most common side effects seen in clinical trials were nausea, vomiting, diarrhea, headache, decreased appetite, dizziness, abdominal pain, itching, drowsiness, and elevated levels of liver enzymes (transaminases) and creatinine.1 Miltefosine is not recommended for pregnant women; this drug bears a black box warning highlighting the harm it could cause to a fetus. The FDA therefore strongly urges healthcare professionals to advise women to use effective contraception during and for five months after miltefosine therapy.1
Although leishmaniasis is prevalent in tropical areas, it is reassuring to know that there is a treatment available here if necessary. The release of drugs to treat diseases that are rare in the U.S. elucidates the relentless efforts of researchers and the FDA.
SOURCES:
- FDA approves Impavido to treat tropical disease leishmaniasis. U.S Food and Drug Administration. March 19, 2014. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm389671.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed May 20th, 2014
- Paladin Announces Extension of the Impavido® PDUFA Date by Three Months. Paldin. November 11th, 2013. http://paladin-labs.mwnewsroom.com/press-releases/paladin-announces-extension-of-the-impavidor-pdu-tsx-plb-201311110910580001. Accessed May 20th, 2014
- Griwank K, Gazeau C, Eichorn A, Stebut E. Miltefosine efficiently eliminates leishmania major Amastigotes from Infected Murine Dendtritic Cells without Altering Their Immune Functions. Antimicrobial Agents and Chemotherapy. http://aac.asm.org/content/54/2/652.long. Accessed May 29th, 2014.
- U.S FDA approves Paladin drug to treat tropical disease. Reuters. March 19, 2014. http://www.reuters.com/article/2014/03/19/fda-leishmaniasis-idUSL2N0MG23020140319. Accessed May 20th, 2014
[pubmed_related keyword1=”PDUFA” keyword2=”tropical” keyword3=”disease”]
