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miltefosine

Trade name: impavido

Small moleculeapprovedOrphan Drug FDAFast Track FDA

Approved

Mar 19, 2014

Miltefosine is an anti-leishmanial agent. It is an alkyl phospholipids compound, was originally intended for breast cancer and other solid tumors. However, it could not be developed as an oral agent because of dose-limiting gastro-intestinal toxicity, and only a topical formulation is approved for skin metastasis. But Miltefosine showed excellent antileishmanial activity both in vitro and in experimental models. Miltefosine is effective in vitro against both promastigotes and amastigotes of various species of Leishmania and also other kinetoplastidae (Trypanosoma cruzi,T. brucei) and other protozoan parasites (Entamoeba histolytica, Acanthamoeba). Mechanism of action is unknown. It is likely to involve interaction with lipids (phospholipids and sterols), including membrane lipids, inhibition of cytochrome c oxidase (mitochondrial function), and apoptosis-like cell death. Miltefosine is approved for the treatment of Visceral leishmaniasis (due to Leishmania donovani), Cutaneous leishmaniasis (due to Leishmania braziliensis, Leishmania guyanensis, and Leishmania panamensis) and Mucosal leishmaniasis (due to Leishmania braziliensis). — NCATS

Clinical trial activity

45 trials · 56 clinical orgs · 1 marketing orgs

Phase 1
5
Phase 2
37
Phase 3
15
Phase 4
2

Earliest trial started Apr 1, 2004 (NCT00373776)

Timeline

2000s

  1. Jan 1, 2004

    Earliest Phase 1 Sponsor(trial)

  2. Jan 1, 2005

    Earliest Phase 2 Sponsor(trial)

  3. Jan 1, 2006

    Earliest Phase 3 Sponsor(trial)

  4. Oct 10, 2006

    Orphan Drug Designation

2010s

  1. Mar 19, 2014

    Knight Therapeutics — Earliest FDA Approval

  2. Mar 19, 2014

    Knight Therapeutics — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

  • Unspecified targetOTHER

    Unknown

    The mechanism of action of miltefosine is likely to involve interaction with lipids (phospholipids and sterols), including membrane lipids, inhibition of cytochrome c oxidase (mitochondrial function), and apoptosis-like cell death.

Approval history

  • approvedPriority reviewFast trackMar 19, 2014

Chemistry & pharmacology

Loading structure…

SMILES

CCCCCCCCCCCCCCCCOP([O-])(=O)OCC[N+](C)(C)C
Mol. weight
407.568 g/mol
Lipinski Ro5
1 violation(s)
Rule of 3
No
Chirality
Racemic Mixture
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

Black box warningFirst in class

Sources

Also known as

  • d18506
  • d 18506
  • d-18506
  • d-18506
  • d-18506
  • ethanaminium, 2-(((hexadecyloxy)hydroxyphosphinyl)oxy)-n,n,n-trimethyl-, hydroxide, innner salt
  • hdpc
  • hdpc
  • hdpc
  • hdpc
  • hexadecyl 2-(trimethylazaniumyl)ethyl phosphate
  • hexadecyl 2-(trimethylazaniumyl)ethyl phosphate
  • hexadecylphosphocholine
  • hexadecylphosphocholine
  • hexadecylphosphocholine
  • hexadecylphosphocholine
  • hexadecylphosphorylcholine
  • hexadecylphosphorylcholine
  • hexadecylphosphorylcholine
  • impavido
  • impavido
  • impavido
  • impavido
  • impavido
  • impavido
  • impavido
  • miltefos
  • miltefosin
  • miltefosin
  • miltefosina
  • miltefosina
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltefosine
  • miltéfosine
  • miltéfosine
  • miltex
  • miltex
  • miltex
  • mitefosine
  • monohexadecylphosphocholine
  • monohexadecylphosphocholine
  • monohexadecylphosphorylcholine
  • monohexadecylphosphorylcholine
  • n-hexadecylphosphorylcholine
  • tf 002
  • tf 002

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT07149753Phase 1Jan 1, 2026Fundación Oftalmológica de Santander Clínica Carlos Ardila Lulle, Universidad Industrial de Santander
NCT06997159Phase 2Nov 16, 2025Drugs for Neglected Diseases Initiative, Novartis
NCT06550609Phase 2Oct 31, 2024Fundacion Nacional de Dermatologia
NCT06251739Early Phase 1 (Phase 1)Oct 30, 2023International Center for Diarrheal Disease Research
NCT06040489Phase 2/Phase 3 (Phase 3)Jun 22, 2022University of Brasilia
NCT04799236Phase 3Apr 1, 2021AB Foundation for Medical Research, Centro Nacional de Enfermedades Tropicales CENETROP, Fundacion Nacional de Dermatologia, Hospital Dermatológico de Jorochito
NCT04515186Phase 3Nov 9, 2020Cayetano Heredia University, Drugs for Neglected Diseases Initiative, Federal University of Bahia, Federal University of Mato Grosso do Sul, Fundacion Nacional de Dermatologia, Instituto Conmemorativo Gorgas de Estudios de la Salud
NCT04268524Phase 3Oct 10, 2020Medecins Sans Frontieres
NCT03829917Phase 2/Phase 3 (Phase 3)Feb 28, 2019Fundacion Nacional de Dermatologia, Government of Bolivia, Hospital Dermatológico de Jorochito, The Alfred Berman Foundation for Medical Research
NCT03399955Phase 2May 9, 2018Drugs for Neglected Diseases Initiative
NCT03129646Phase 3Aug 1, 2017Drugs for Neglected Diseases Initiative, Institute of Endemic Diseases, Sudan, Kenya Medical Research Institute, Makerere University, Netherlands Cancer Institute, University of Gondar, University of Khartoum
NCT03023111Phase 3Mar 1, 2017Federal University of Bahia, Instituto de Medicina Integral Professor Fernando Figueira, Oswaldo Cruz Foundation
NCT02687971Phase 2Dec 1, 2016Cayetano Heredia University, Drugs for Neglected Diseases Initiative, Universidad de Antioquia
NCT02730117N/AMar 1, 2016Chulalongkorn University
NCT03445897Phase 2Jan 31, 2016AB Foundation for Medical Research
NCT02530697Phase 2Aug 1, 2015University of Brasilia
NCT02431143Phase 2May 1, 2015Drugs for Neglected Diseases Initiative, Kenya Medical Research Institute
NCT02011958Phase 3Jul 1, 2014Addis Ababa University, Drugs for Neglected Diseases Initiative, Institute of Tropical Medicine, Belgium, Medecins Sans Frontieres, Slotervaart Hospital, University of Gondar, University of London
NCT02193022Phase 3Jul 1, 2014International Center for Diarrheal Disease Research, Thrasher Research Fund
NCT01975051Phase 4Jan 1, 2013International Center for Diarrheal Disease Research, University of Nagasaki
Showing 20 of 45 trials
Page 1 / 3

Organizations

Research & Development (56)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Drugs for Neglected Diseases InitiativeAcademic/Hospital111022007
AB Foundation for Medical ResearchAcademic/Hospital9622004
Fundacion Nacional de DermatologiaAcademic/Hospital4322019
International Center for Diarrheal Disease ResearchAcademic/Hospital4332010
Banaras Hindu UniversityAcademic/Hospital3322006
Federal University of BahiaAcademic/Hospital3222007
Kenya Medical Research InstituteAcademic/Hospital3022010
University of BrasiliaAcademic/Hospital3322009
AEterna ZentarisFor profit2012005
Cayetano Heredia UniversityAcademic/Hospital2022016
Centro Internacional de Entrenamiento e Investigaciones MédicasAcademic/Hospital2222007
Foundation FaderAcademic/Hospital2212007
Hospital Dermatológico de JorochitoAcademic/Hospital2012019
Humboldt UniversityAcademic/Hospital2112007
Medecins Sans FrontieresAcademic/Hospital2112014
Rajendra Memorial Research Institute of Medical SciencesAcademic/Hospital2022007
Universidad de AntioquiaAcademic/Hospital2122006
University of GondarAcademic/Hospital2012014
World Health OrganizationGovernment2022006
Addis Ababa UniversityAcademic/Hospital1012014
56 organizations
Page 1 / 3

Marketing (1)

OrganizationOrg typeRelationshipDate
Knight TherapeuticsFor profitNDAMar 19, 2014