Miltefosine 50 mg, 100 capsules: profile, composition and research

Reference page for Miltefosine 50 mg, 100 capsules. Active substance: Милтефосин / Miltefosine.

Key facts

What miltefosine is

Miltefosine is an antiparasitic medicine used against diseases caused by protozoa of the genus Leishmania. Its main advantage is that it was the first effective treatment for visceral leishmaniasis that is taken by mouth rather than given intravenously.

Leishmaniasis is spread by sandfly bites and occurs in tropical and subtropical regions. It takes several forms: cutaneous, where the skin is affected, and visceral, where internal organs are involved. There is also a mucosal form that affects the tissues of the nose and mouth.

Chemically, miltefosine is a derivative of alkylphosphocholine. It was first studied as an anticancer substance, but its practical use was found in parasitology.

How miltefosine works

Effect on the membrane

The drug interferes with the metabolism of the phospholipids that build the parasite's envelope. The membrane stops working properly and the parasite cell loses its internal balance.

Death of the parasite

Damage to the envelope triggers a process similar to programmed cell death. The parasite does not multiply and dies inside the host.

Effect on signalling

Miltefosine also touches the signalling machinery of the parasite cell. That is why it is sometimes studied in combinations: different points of attack reduce the chance that the parasite survives.

Approved uses

The prescribing information lists leishmaniasis in its main forms: visceral, cutaneous and mucosal. Separately, miltefosine is used for a very rare and severe brain infection caused by free-living amoebae (primary amoebic meningoencephalitis).

The dose is calculated from body weight and the form of the disease, so treatment is always individual. Miltefosine is taken with food: this improves absorption and reduces stomach discomfort.

The medicine has a serious effect on the body, so it is used under medical supervision and with laboratory monitoring. Self-treatment is especially dangerous here.

Published research

Primary sources, verified against PubMed:

• Treatment of New World Mucosal Leishmaniasis: Randomized Comparison of Glucantime, Liposomal Amphotericin B, and Miltefosine, PubMed 41818822 (Am J Trop Med Hyg, 2026).
• Efficacy of miltefosine in the treatment of visceral leishmaniasis in India after a decade of use, PubMed 22573856 (Clin Infect Dis, 2012).
• Safety and efficacy of paromomycin/miltefosine/liposomal amphotericin B combinations for the treatment of post-kala-azar dermal leishmaniasis in Sudan: a phase II, open label, randomized, parallel arm study, PubMed 37988402 (PLoS Negl Trop Dis, 2023).

Listed for reference. Publication of a study is not a claim about this particular product.

Precautions

• This is a prescription medicine. Prescribing and monitoring belong to a physician.
• Miltefosine must not be used in pregnancy: it can cause severe birth defects. A confirmed negative pregnancy test is required before treatment starts.
• Reliable contraception is needed during treatment and for the period a doctor specifies afterwards.
• Breastfeeding is stopped for the duration of treatment.
• Nausea, vomiting, diarrhoea, abdominal pain, dizziness and loss of appetite are possible. Marked symptoms should be reported to a doctor.
• Yellowing of the skin or eyes, dark urine, severe abdominal pain, hearing changes or swelling require urgent medical help.

Frequently asked