Miltefosine was initially approved by the United States Food and Drug Administration (FDA) on March 19, 2014. 

A 2021 study found that miltefosine, when taken orally, effectively kills the parasite responsible for leishmaniasis by blocking key pathways. The study also found that it works against visceral, cutaneous, and mucosal forms of the disease.

Miltefosine treats leishmaniasis, a parasitic disease transmitted by sandflies, including the most severe form, kala-azar (visceral leishmaniasis), which affects internal organs. Miltefosine is indicated for adults and adolescents aged 12 years and older who weigh at least 30 kg (66 lbs) for the treatment of visceral leishmaniasis caused by Leishmania donovani and cutaneous leishmaniasis due to Leishmania braziliensis. 

It also effectively treats different forms of leishmaniasis, including cutaneous, visceral, and mucosal types. It also targets infections caused by free-living amoebas like Naegleria fowleri and Balamuthia mandrillaris.

Miltefosine should be taken orally with food to help reduce gastrointestinal side effects. If you weigh between 30 and 44 kg, take one 50 mg capsule twice daily for 28 consecutive days. If you weigh 45 kg or more, take one 50 mg capsule three times daily for the same duration. Follow this dosing schedule closely to ensure effective treatment for leishmaniasis. Regularly check in with your doctor to monitor your response and address any potential side effects.

Common side effects of Miltefosine may include:

• Nausea & Vomiting
• Diarrhea
• Abdominal pain
• Decreased appetite
• Dizziness
• Headache
• Sleepiness
• Skin itching
• Abnormalities in liver or kidney tests

Serious side effects of Miltefosine include:

• Fertility problems
• Testicular pain and absent or decreased ejaculation
• Kidney and liver problems
• Serious skin problems (e.g., Stevens-Johnson Syndrome)
• Decrease in platelets

The warnings of Miltefosine are as follows: 

• Fertility concerns: Miltefosine may cause fertility problems in animals, and you should be aware that its effects on human fertility are unclear.
• Serious skin reactions: Watch for signs of Stevens-Johnson Syndrome, a severe skin condition. Seek immediate medical attention if you develop a rash with blisters.
• Kidney and liver issues: Miltefosine may lead to kidney and liver problems. Your healthcare provider should regularly monitor your liver and kidney function during treatment.
• Gastrointestinal effects: You may experience vomiting, diarrhoea, and dehydration. Stay hydrated and contact your healthcare provider if severe symptoms occur.
• Effects on contraception: Miltefosine may decrease the effectiveness of oral contraceptives. Consider using an additional form of birth control during treatment.
• Blood cell counts: Miltefosine can lower your platelet count, which is essential for blood clotting. Your healthcare provider may recommend regular blood tests to monitor this.
• Monitoring and reporting: Report any side effects to your healthcare provider and attend regular check-ups to ensure your safety and the effectiveness of the treatment.

Before starting miltefosine, you should consider several important precautions. Avoid using this medication if you are pregnant or planning to become pregnant, as it can harm the fetus. Use effective contraception during treatment. Discuss potential fertility effects with your healthcare provider if you plan to conceive. If you have kidney or liver issues, inform your doctor, as they may need to monitor your condition closely. 

Be aware that miltefosine can cause gastrointestinal problems; stay hydrated to avoid these effects. Watch for any skin reactions and report them immediately. Additionally, provide your healthcare provider with a complete list of all medications you are taking to avoid potential interactions. Regular follow-ups are required to make sure the treatment is effective and to manage any side effects.

Inform your doctor if you have a history of kidney or liver issues, as they may require close monitoring during treatment. Regularly check for any gastrointestinal symptoms and stay hydrated to prevent dehydration. Report any skin reactions or symptoms of low blood cell counts to your healthcare provider immediately. Always disclose all other medications you are taking to avoid potential interactions. Use miltefosine during pregnancy only when necessary and monitor for any allergic reactions; report any concerns without delay.

1. Efficacy and Tolerability of Miltefosine in the Treatment of Cutaneous Leishmaniasis - PMC (nih.gov)
2. https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/204684s000lbl.pdf
3. https://ncvbdc.mohfw.gov.in/WriteReadData/l892s/Guidelines_on_miltefosine.pdf
4. https://pubmed.ncbi.nlm.nih.gov/10588964/https://pubmed.ncbi.nlm.nih.gov/10588964/

The drug information on this page is not a substitute for medical advice. It is meant for educational purposes only. For further details, consult your doctor about your medical condition to know if you can receive this treatment.