Miltefosine 50mg capsules
Requires a prescription from a doctor or prescriber
Miltefosine is a broad spectrum antimicrobial, anti-leishmanial, phospholipid drug that was originally developed in the 1980s as an anti-cancer agent.
Safety information for pregnancy and breastfeeding
Pregnancy
Always consult your doctor or midwife before taking any medicine during pregnancy or while breastfeeding. Source: DrugBank (CC BY-NC 4.0).
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Yellow Card reports
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Submit a Yellow Card report to the MHRA
Data from the MHRA Yellow Card scheme. A reported reaction does not necessarily mean the medicine caused it. Contains public sector information licensed under the Open Government Licence v3.0.
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Suspected adverse reactions reported for Miltefosine
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EudraVigilance data is published by the European Medicines Agency (EMA). A suspected adverse reaction is not necessarily caused by the medicine.
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Therapeutically similar medicines
Tablets & capsules
(1)Injectables
(1)Similarity is based on WHO Anatomical Therapeutic Chemical (ATC) classification and on a factual NHS dm+d therapeutic-grouping code prefix. Source data: NHS dm+d via TRUD (OGL v3.0), WHO ATC/DDD Index.
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Codes for healthcare professionals and prescribing systems
These codes are used by healthcare IT systems and prescribers to identify this medicine.
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SNOMED CT and dm+d codes from NHS TRUD (Technology Reference data Update Distribution), licensed under the Open Government Licence v3.0. ATC codes from the WHO Collaborating Centre for Drug Statistics Methodology (whocc.no).
Active and completed clinical studies from ClinicalTrials.gov
Source: ClinicalTrials.gov, a database of the U.S. National Library of Medicine (NLM), National Institutes of Health (NIH). Data accessed via ClinicalTrials.gov API v2. Trial information is provided for research purposes and does not constitute medical advice.
Academic studies and reviews for this medicine's active substance
Showing the 50 most relevant studies.
Reviews & meta-analyses: 15 · Randomised trials: 11 · 2006–2026
Showing the 50 most relevant studies, sorted by most relevant.
Abbasi E
2025
BackgroundVisceral leishmaniasis (VL), also known as kala-azar, is a life-threatening neglected tropical disease primarily caused by Leishmania donovani and transmitted by infected sandflies. Despite notable advancements in public health, VL continues to pose significant challenges, especially in South Asia, East Africa, and South America.MethodsA systematic review was conducted in accordance with PRISMA guidelines. Peer-reviewed literature published from 2000 to 2024 was retrieved from PubMed, Scopus, and Web of Science to assess epidemiological patterns, diagnostic advancements, therapeutic options, and vector control strategies.ResultsThe analysis revealed progress in diagnostic tools such as rK39-based rapid tests and molecular diagnostics. Therapeutic improvements, notably liposomal amphotericin B and miltefosine, have emerged, although drug resistance and limited accessibility remain problematic. Vector control through residual spraying and community programs shows promise but is hindered by insecticide resistance. Furthermore, HIV co-infection and climate-driven spread exacerbate control efforts.ConclusionsComprehensive management of VL requires integrative approaches that combine novel diagnostics, targeted treatment, innovative vector control, and robust public health systems. Emphasis on vaccine development, digital health solutions, and community participation is crucial for sustainable control and eventual elimination.
Abstract licence: CC BY
Ijlal A, Farooqi B, Kanwal H, et al.
2026
Background: and Objectives: Balamuthia mandrillaris granulomatous amoebic encephalitis (GAE) is a rare, frequently fatal central nervous system infection in which delayed recognition remains a major determinant of outcome. The increasing use of metagenomic next-generation sequencing (mNGS), plasma microbial cell-free DNA sequencing and targeted sequencing has changed the diagnostic pathway for unexplained encephalitis, but the survival implications of earlier molecular diagnosis remain unclear. Materials and Methods: A systematic review of peer-reviewed reports published from January 2015 to June 2026 was designed according to PRISMA 2020. Eligible records reported human B. mandrillaris GAE, encephalitis, meningoencephalitis or encephalomyelitis with extractable patient-level diagnostic, treatment or outcome information. Data were narratively synthesised because case definitions, sample types, timing of testing and follow-up were heterogeneous. Results: The evidence base consisted mainly of case reports, small case series and retrospective clinical summaries. Across recent reports, B. mandrillaris GAE commonly mimicked malignancy, tuberculosis, fungal infection, autoimmune encephalitis or bacterial brain abscess. Diagnostic delay was driven by nonspecific symptoms, ring-enhancing lesions, negative routine cultures and limited clinician familiarity with free-living amoebae. Molecular methods, particularly mNGS/NGS applied to cerebrospinal fluid, plasma, blood, brain tissue or bronchoalveolar lavage fluid, repeatedly enabled antemortem diagnosis when routine tests were unrevealing. Survival reports shared a pattern of earlier tissue diagnosis or molecular identification, neurosurgical intervention when feasible, and prolonged multidrug therapy, sometimes including miltefosine or nitroxoline. Conclusions: B. mandrillaris GAE should be considered in subacute encephalitis with mass-like or multifocal enhancing lesions and negative conventional investigations. Early biopsy or non-invasive sequencing, followed by confirmatory testing and expert-guided combination therapy, represents the most actionable strategy to reduce diagnostic delay.
Abstract licence: CC BY
Sarantopoulos A, Quattrocchi A, Kopsidas I, et al.
2026
Background/objectivesPrimary amoebic meningoencephalitis (PAM) is a rare, fulminant, and often fatal central nervous system infection caused by the opportunistic free-living amoeba Naegleria fowleri. Although Naegleria species are widely present in freshwater and soil worldwide, human disease is associated specifically with pathogenic N. fowleri rather than the many nonpathogenic environmental species, and virulence may vary across N. fowleri isolates. This systematic review aimed to synthesize contemporary global data from 2000 to 2024 to identify recent trends in epidemiology, clinical presentation, diagnosis, treatment, and outcomes.MethodsA systematic literature search was conducted across PubMed, Scopus, and the Cochrane Library, identifying 58 eligible publications encompassing 66 individual cases.ResultsMost reports originated from the United States, India, and China. The median patient age was 14 years, with 78% of cases occurring in males. Annual case reports increased from one per year (2000-2005) to over four per year (2020-2024), reflecting either a true rise in incidence or improved detection. Common presenting symptoms included fever, headache, and altered mental status. Diagnosis was confirmed via polymerase chain reaction (PCR) testing or post-mortem biopsy in nearly one-third of cases. Treatment regimens varied, with amphotericin B and miltefosine being the most frequently used agents. Overall mortality was 83%, with survival strongly associated with early initiation of combination therapy. Pediatric patients had a higher survival rate (22%) compared to adults (7.1%).ConclusionsThe findings highlight the need for heightened clinical awareness, especially in the context of climate-driven ecological changes that may expand N. fowleri's geographic range. This review underscores critical gaps in surveillance and diagnostics and emphasizes the importance of a One Health approach to addressing emerging threats like PAM. Further research into novel therapeutics, rapid diagnostics, and global case reporting systems is urgently needed.
Abstract licence: CC BY
Vieira ACP, Lins FC, Baquião AC
2026
- Leishmaniasis, Cutaneous
- Meglumine
- Phosphorylcholine
BackgroundCutaneous leishmaniasis (CL) affects up to 1.2 million people annually, mainly in resource-limited regions. Meglumine antimoniate, the standard treatment, is limited by systemic toxicity, injectable administration, and increasing resistance. Miltefosine, an oral alternative, offers practical advantages, although comparative efficacy and safety data remain inconsistent.ObjectiveTo compare the efficacy and safety of miltefosine versus meglumine antimoniate for New World CL.MethodsThe authors systematically searched PubMed, Embase, Scopus, and the Cochrane Library for randomized controlled trials directly comparing miltefosine and meglumine antimoniate. Risk Ratios (RRs) with 95% Confidence Intervals (95% CIs) were calculated using random-effects models. Heterogeneity was assessed with the I² statistic. Risk of bias was evaluated using the Cochrane RoB-2 tool. Certainty of evidence was assessed using the Grading of Recommendations, Assessment, Development, and Evaluations (GRADE) approach.ResultsEight trials involving 898 patients (502 treated with miltefosine, 396 with meglumine antimoniate) were included. Miltefosine showed significantly higher cure rates at two months (RR = 0.83; 95% CI: 0.71-0.98; I2 = 0%). Differences at six months were not statistically significant. Gastrointestinal side effects were more frequent with miltefosine, whereas hepatic enzyme elevations, arthralgia (RR = 10.08; 95% CI: 2.36-43.12), and fever (RR = 2.98; 95% CI: 1.53-5.80) were more common with meglumine antimoniate.Study limitationsHigh heterogeneity, short follow-up, small sample sizes, and interstudy variability may limit precision.ConclusionMiltefosine shows superior early response and a safer systemic profile. However, the certainty of evidence, as assessed by GRADE, ranged from very low to high across outcomes, and long-term data remain limited, highlighting the need for further high-quality studies with extended follow-up.
Abstract licence: CC BY
Semra Palić, P. Bhairosing, J. Beijnen, et al.
Antimicrobial Agents and Chemotherapy, 2019
S. Iranpour, A. Hosseinzadeh, A. Alipour
Epidemiology and Health, 2019
T. Sunyoto, J. Potet, M. Boelaert
BMJ Global Health, 2018
E. Diro, S. Blesson, T. Edwards, et al.
PLoS Neglected Tropical Diseases, 2019
R. Rahman, Vishal Goyal, R. Haque, et al.
PLoS Neglected Tropical Diseases, 2017
A. Musa, J. Mbui, R. Mohammed, et al.
Clinical Infectious Diseases: An Official Publication of the Infectious Diseases Society of America, 2022
Sources: aggregated from Europe PMC (EMBL-EBI), OpenAlex, Crossref, PubMed and other open scholarly databases. Retracted articles are excluded. Study information is provided for research purposes and does not constitute medical advice.
Pharmacology and chemical data from DrugBank
Key facts
Drug status
Approved
Major interactions
None known
Half-life
7.05 days
Mechanism
Miltefosine has demonstrated activity against Leishmania parasites and neoplasti…
Food interactions
2 warnings
Human targets
2 targets
Data: DrugBank · CC BY-NC 4.0
Pharmacokinetics at a glance
Absorption
82%
Half-life
7.05 days
Protein binding
96%
Volume of distribution
Metabolism
Elimination
Clearance
159 hours
Pharmacokinetic data: DrugBank · CC BY-NC 4.0
Known interactions with other medicines. Always consult a healthcare professional.
Showing 50 of 52 interactions
Stevens-Johnson syndrome has been reported, therefore therapy should be discontinued if an exfoliative or bullous rash occurs during treatment.
How the body processes this drug — absorption, distribution, metabolism, and elimination
Proteins and enzymes this drug interacts with in the body
PMID:10681567 PMID:1420353 PMID:17603006
Hydrolyzes the ester bond of the fatty acyl group attached at sn-2 position of phospholipids (phospholipase A2 activity) with preference for phosphatidylethanolamines and phosphatidylglycerols over phosphatidylcholines .
PMID:10681567 PMID:1420353 PMID:17603006
May play a role in the biosynthesis of N-acyl ethanolamines that regulate energy metabolism and inflammation in the intestinal tract. Hydrolyzes N-acyl phosphatidylethanolamines to N-acyl lysophosphatidylethanolamines, which are further cleaved by a lysophospholipase D to release N-acyl ethanolamines (By similarity). May act in an autocrine and paracrine manner .
PMID:25335547 PMID:7721806
Upon binding to the PLA2R1 receptor can regulate podocyte survival and glomerular homeostasis .
PMID:25335547
Has anti-helminth activity in a process regulated by gut microbiota.
Upon helminth infection of intestinal epithelia, directly affects phosphatidylethanolamine contents in the membrane of helminth larvae, likely controlling an array of phospholipid-mediated cellular processes such as membrane fusion and cell division while providing for better immune recognition, ultimately reducing larvae integrity and infectivity (By similarity)
PMID:2897240 PMID:35970996 PMID:8898203 PMID:9038218 PMID:35507548
Catalyzes the flop of phospholipids from the cytoplasmic to the exoplasmic leaflet of the apical membrane. Participates mainly to the flop of phosphatidylcholine, phosphatidylethanolamine, beta-D-glucosylceramides and sphingomyelins .
PMID:8898203
Energy-dependent efflux pump responsible for decreased drug accumulation in multidrug-resistant cells PMID:2897240 PMID:35970996 PMID:9038218
Enzymes involved in drug metabolism — important for understanding drug interactions
Proteins that transport this drug across cell membranes
PMID:2897240 PMID:35970996 PMID:8898203 PMID:9038218 PMID:35507548
Catalyzes the flop of phospholipids from the cytoplasmic to the exoplasmic leaflet of the apical membrane. Participates mainly to the flop of phosphatidylcholine, phosphatidylethanolamine, beta-D-glucosylceramides and sphingomyelins .
PMID:8898203
Energy-dependent efflux pump responsible for decreased drug accumulation in multidrug-resistant cells PMID:2897240 PMID:35970996 PMID:9038218
ATC P01CX04
Chemical identifiers
CAS, UNII, InChI Key and database cross-references
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Chemical identifiers
CAS, UNII, InChI Key and database cross-references
Linked compound data from DrugBank Open Data (CC BY-NC 4.0)
Miltefosine
Additional database identifiers
ChemSpider
3473
BindingDB
50034220
HUGO Gene Nomenclature Committee (HGNC)
HGNC:9030
GeneCards
PLA2G1B
GenBank Gene Database
M21054
GenBank Protein Database
190013
Guide to Pharmacology
1416
UniProt Accession
PA21B_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:40
GenAtlas
ABCB1
GeneCards
ABCB1
GenBank Gene Database
M14758
GenBank Protein Database
307180
Guide to Pharmacology
768
UniProt Accession
MDR1_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:9067
GenAtlas
PLD1
GeneCards
PLD1
GenBank Gene Database
U38545
GenBank Protein Database
1185463
Guide to Pharmacology
1433
UniProt Accession
PLD1_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:40
GenAtlas
ABCB1
GeneCards
ABCB1
GenBank Gene Database
M14758
GenBank Protein Database
307180
Guide to Pharmacology
768
UniProt Accession
MDR1_HUMAN
DrugBank citations
If you use DrugBank data in your research, please cite:
- DrugBank 6.02024Recommended citationKnox C., Wilson M., Klinger C.M., et alDrugBank 6.0: the DrugBank Knowledgebase for 2024Nucleic Acids Res. 2024 Jan 552(D1):D1265-D1275
- DrugBank 5.02018Wishart D.S., Feunang Y.D., Guo A.C., et alDrugBank 5.0: a major update to the DrugBank database for 2018Nucleic Acids Res. 2017 Nov 846(D1):D1074-D1082
- DrugBank 4.02014Law V., Knox C., Djoumbou Y., et alDrugBank 4.0: shedding new light on drug metabolismNucleic Acids Res. 2014 Jan 142(1):D1091-7
- DrugBank 3.02011Knox C., Law V., Jewison T., et alDrugBank 3.0: a comprehensive resource for 'omics' research on drugsNucleic Acids Res. 2011 Jan39(Database issue):D1035-41
- DrugBank 2.02008Wishart D.S., Knox C., Guo A.C., et alDrugBank: a knowledgebase for drugs, drug actions and drug targets.Nucleic Acids Research2008 Jan36(Database issue):D901-6
- DrugBank 1.02006Wishart D.S., Knox C., Guo A.C., et alDrugBank: a comprehensive resource for in silico drug discovery and exploration.Nucleic Acids Research2006 Jan 134(Database issue):D668-72