Miltefosine

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Cat Number

API0232200

CAS Number

58066-85-6

For research use only. We do not supply to patients.

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CAS Number

58066-85-6

EINECS

622-572-6

Storage

Store at room temperature

Synonyms

Hexadecylphosphocholine; Impavido; Miltex; HDPC; Miltefosina; Miltefosinum

Molecular Formula

C21H46NO4P

Molecular Weight

407.6

Smiles

CCCCCCCCCCCCCCCCOP(=O)([O-])OCC[N+](C)(C)C

Appearance

White to off-white solid

Melting Point

232-234°C (dec.)

General Description

Miltefosine is a synthetic alkylphosphocholine with a tetradecyl chain, structurally unrelated to other antiparasitic agents. It is a zwitterionic compound with amphiphilic properties, allowing it to interact with cell membranes and disrupt phospholipid metabolism.

Mechanism of Action

Miltefosine acts by interfering with the phospholipid metabolism of the parasite, inhibiting the synthesis of phosphatidylcholine and disrupting the integrity of the cell membrane. It also inhibits the Akt/PKB signaling pathway, leading to apoptosis of the parasite. The drug has a direct effect on the mitochondrial membrane of leishmania, causing depolarization and cell death. Its multi‑target mechanism contributes to its activity against resistant strains.

Application

Miltefosine is indicated for the treatment of visceral leishmaniasis (Kala‑azar) and for cutaneous and mucosal leishmaniasis. It is effective against both Leishmania donovani and L. infantum. The drug is also used for the treatment of naegleria and other free‑living amebic infections.

Miltefosine suppressed human eosinophil effector functions in vitro (CD11b up-regulation, degranulation, chemotaxis, downstream signaling) and reduced immune cell infiltration in mouse models of allergic migration. In an ovalbumin-induced allergic lung inflammation model, miltefosine improved lung function parameters. Miltefosine shows strong immunomodulatory activity against eosinophilic inflammation, suggesting potential for allergic diseases.

Fig. 1 Miltefosine concentration‐dependently inhibits eosinophil activation. (Knuplez E, et al., 2021)

References

Knuplez E, et al. The anti-parasitic drug miltefosine suppresses activation of human eosinophils and ameliorates allergic inflammation in mice. Br J Pharmacol. 2021;178(5):1234-1248.

In a phase 3 open‑label trial (439 patients with primary visceral leishmaniasis in eastern Africa), paromomycin + miltefosine (PM/MF, 14 days) showed 91.2% definitive cure at 6 months vs 91.8% for sodium stibogluconate + paromomycin (SSG/PM, 17 days). Noninferiority was narrowly missed in modified ITT (difference 0.6%; 97.5% CI ‑6.2 to 7.4) but achieved in per‑protocol analysis. PM/MF is more patient‑friendly with fewer injections, shorter duration, and no SSG cardiotoxicity.

Fig. 2 Patient disposition. (Musa AM, et al., 2023)

References

Musa AM, et al. Paromomycin and Miltefosine Combination as an Alternative to Treat Patients With Visceral Leishmaniasis in Eastern Africa: A Randomized, Controlled, Multicountry Trial. Clin Infect Dis. 2023;76(3):e1177-e1185.

Does Miltefosine require protection from light and moisture during storage?

Yes, it is sensitive to both light and moisture. Light causes photodegradation and moisture promotes hydrolysis of the phosphate ester. Store in light-resistant, tightly sealed containers with desiccant.

What is the recommended storage temperature for Miltefosine?

Store at controlled room temperature (15-25°C). Avoid excessive heat above 30°C, which can accelerate oxidation and decomposition.

Is Miltefosine stable in solution for oral and topical use?

Aqueous solutions are stable when protected from light and stored at room temperature.

How is the impurity miltefosine hexadecanol (a hydrolysis product) monitored?

This degradation product is quantified using a stability-indicating HPLC method with mass spectrometric detection, ensuring it remains within ICH limits.