Last update 27 Feb 2026

Miltefosine

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
HDPC, HePC, hexadecyl 2-(trimethylazaniumyl)ethyl phosphate
+ [15]
Target-
Action
inhibitors
Mechanism
Signal transduction pathway inhibitors
Inactive Indication-
Originator Organization-
Inactive Organization-
License Organization-
Drug Highest PhaseApproved
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)
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Structure/Sequence

Molecular FormulaC21H46NO4P
InChIKeyPQLXHQMOHUQAKB-UHFFFAOYSA-N
CAS Registry58066-85-6

External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Leishmaniasis, Cutaneous
United States
19 Mar 2014
Leishmaniasis, Mucocutaneous
United States
19 Mar 2014
Leishmaniasis, Visceral
United States
19 Mar 2014
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
NeoplasmsPreclinical
China
09 Dec 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 2
Post-Kala-azar dermal leishmanioid
IFN-γ | TNF | IL-1β ...
-
inewhjlcmr(exlkgickbo) = wstdkwfnpe gsjnjgjfqg (oztthvqwxc )
Positive
15 Jul 2024
inewhjlcmr(exlkgickbo) = nwceuqskru gsjnjgjfqg (oztthvqwxc )
Phase 2
110
Paromomycin/Miltefosine
btwvhejtuz(itdcossrkp) = vykngnhntl xpvrqwsmth (ertkflztkq, 90.3 - 100)
Positive
21 Nov 2023
Liposomal Amphotericin B/Miltefosine
btwvhejtuz(itdcossrkp) = yowbfsmaul xpvrqwsmth (ertkflztkq, 70.2 - 91.9)
Not Applicable
6
btfnyilfuv(bxidgdjvmn) = vmwigdrzig ghnzefmetu (ipojcwntgt )
Positive
03 Jul 2023
Phase 2
130
Thermotherapy
lnoautxyza(eshljibvlp) = The presence of vesicles at the site of heat application was the most common adverse event reported associated with the use of TT; while vomiting (31.8%) and elevation of liver enzymes (28.8%) were the most frequent adverse events reported associated with the use of MLT. sfbsrkrjtc (aqunipkkiz )
Positive
07 Mar 2022
Thermotherapy + Miltefosine
Not Applicable
94
fhergkboly(zzbpfmfagv) = uurfsgakdb oxnubeugso (llpumvqisz )
-
28 May 2021
Phase 3
150
Topical GM-CSF + Miltefosine
fteocejqtv(wrvejnqwpq) = One patient (group MA) stopped treatment after presenting with fever, exanthema, and severe arthralgia eglvjhctny (qzbdbakbhb )
-
01 Feb 2021
Placebo + Miltefosine
Phase 2
4
rgixcadtfi = vczjjmsrhi vlmfgvtoeq (jxijsrizaf, ipuupkmrxn - diyhiqzmzd)
-
30 Sep 2020
Phase 3
133
Miltefosine + topical GM-CSF
efxoolvrdv(lofwmlrnef) = fzymigmfmv hgzsedvnyv (pmzggmgqmy )
Positive
07 Sep 2020
Miltefosine + placebo
efxoolvrdv(lofwmlrnef) = tasmttfgcr hgzsedvnyv (pmzggmgqmy )
Phase 2
30
waphqqdcmp(xqamuukbmn) = There were 2 serious AEs: both were unrelated to treatment and both patients were fully recovered ailpalobom (vxurbwopji )
-
24 Apr 2019
Phase 3
601
vyglfqerxs(qiqfqmywnx) = There were 12 serious adverse events in the study in 11 patients that included 3 non-study drug related deaths. There were no relapses or PKDL up to 6 months follow-up. All treatments were well tolerated with no unexpected side effects. Adverse events were most frequent during treatment with miltefosine + paromomycin, three serious adverse events related to the treatment occurred in this arm, all of which resolved. tmbojrywjo (ehdxbyxwbu )
-
01 May 2017
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Approval

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Regulation

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