Drugs / Miltefosine
Miltefosine
also known as
hexadecylphosphocholine · Impavido · D-18506 · Miltefosina · Miltefosine cap 10mg and 50mg, Impavido (Zentaris) · Miltex · NSC-605583 · NSC-758968
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | US (FDA) | IMPAVIDO | — | 2014-03-19 | fda.gov ↗ |
Trials 37
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT07149753 | Jan → Jul 2026 overdue | cutaneous leishmaniasis | Universidad Industrial de Santander | Not yet recruiting | No outcome recorded |
| Phase 1/23 trials | ||||||
| Phase 1/2 | NCT00373568 | Apr 2005 → Dec 2007 | leishmaniasis | AB Foundation | Completed | No outcome recorded |
| Phase 1/2 | NCT00378495 | Apr 2005 → Apr 2007 | visceral leishmaniasis | AB Foundation | Terminated | No outcome recorded Stop: Enrollment |
| Phase 1/2 | NCT00373776 | Apr 2004 → May 2006 | mucocutaneous leishmaniasis | AB Foundation | Completed | No outcome recorded |
| Phase 218 trials · 2 met primary | ||||||
| Phase 2 | NCT06550609 | Feb 2025 → Nov 2027 expected | mucocutaneous leishmaniasis | Fundacion Nacional de Dermatologia | Recruiting | No outcome recorded |
| Phase 2 | NCT03399955 | May 2018 → May 2021 | cutaneous leishmaniasis | Drugs for Neglected Diseases | Unknown | No outcome recorded |
| Phase 2 | NCT02687971 | Dec 2016 → Aug 2019 | cutaneous leishmaniasis | Drugs for Neglected Diseases | Completed | Met primary |
| Phase 2 | NCT03445897 | Jan 2016 → Jan 2018 | cutaneous leishmaniasis | Jonathan Berman | Completed | No outcome recorded |
| Phase 2 | NCT02530697 | Aug 2015 → Aug 2025 overdue | leishmaniasis | University of Brasilia | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02431143 | May 2015 → Apr 2016 | visceral leishmaniasis | Drugs for Neglected Diseases | Completed | No outcome recorded |
| Phase 2 | NCT02366884 | Jul 2011 → Dec 2022 | neoplasm | Dr. Frank Arguello Cancer Clinic | Unknown | No outcome recorded |
| Phase 2 | NCT01050907 | May 2010 → Feb 2015 | cutaneous leishmaniasis, mucocutaneous leishmaniasis | Knight Therapeutics (USA) Inc | Completed | No outcome recorded |
| Phase 2 | NCT01067443 | Mar 2010 → Jun 2011 | visceral leishmaniasis | Drugs for Neglected Diseases | Completed | No outcome recorded |
| Phase 2 | NCT01377974 | Jul 2009 → Jun 2013 | mucocutaneous leishmaniasis | Brasilia University Hospital | Completed | No outcome recorded |
| Phase 2 | NCT01170949 | Sep 2008 | chronic urticaria | Marcus Maurer | Terminated | No outcome recorded |
| Phase 2 | NCT00371995 | Oct 2007 → Aug 2009 | visceral leishmaniasis | Banaras Hindu University | Completed | No outcome recorded |
| Phase 2 | NCT01635777 | Jul 2007 → Oct 2009 | — | AB Foundation | Completed | No outcome recorded |
| Phase 2 | NCT00600548 | Jul 2007 → Mar 2009 | cutaneous leishmaniasis | Hospital Universitário Professor Edgard Santos | Completed | Met primary |
| Phase 2 | NCT01380301 | Mar 2007 → Aug 2008 | cutaneous leishmaniasis | Foundation Fader | Terminated | No outcome recorded |
| Phase 2 | NCT00370825 | Sep 2006 → ? | visceral leishmaniasis | Banaras Hindu University | Completed | No outcome recorded |
| Phase 2 | NCT00233545 | Sep 2005 → Sep 2007 | cutaneous leishmaniasis | AB Foundation | Completed | No outcome recorded |
| Phase 2 | NCT00537953 | — | cutaneous leishmaniasis | Centro de Investigaciones Bioclínicas de la Fundación Fader | Unknown | No outcome recorded |
| Phase 2/32 trials | ||||||
| Phase 2/3 | NCT06040489 | Jun 2022 → Jun 2024 | mucocutaneous leishmaniasis | University of Brasilia | Unknown | No outcome recorded |
| Phase 2/3 | NCT03829917 | Feb 2019 → Dec 2020 | cutaneous leishmaniasis | Fundacion Nacional de Dermatologia | Completed | No outcome recorded |
| Phase 311 trials · 2 met primary · 3 missed | ||||||
| Phase 3 | NCT04268524 | Oct 2022 → Dec 2025 overdue | cutaneous leishmaniasis | Medecins Sans Frontieres, Netherlands | Recruiting | No outcome recorded |
| Phase 3 | NCT04515186 | Jan 2021 → Nov 2023 | cutaneous leishmaniasis | Drugs for Neglected Diseases | Completed | No outcome recorded |
| Phase 3 | NCT03129646 | Jan 2018 → Dec 2020 | visceral leishmaniasis | Drugs for Neglected Diseases | Completed | Missed primary |
| Phase 3 | NCT03023111 | Jun 2017 → Aug 2019 | cutaneous leishmaniasis | Hospital Universitário Professor Edgard Santos | Completed | Missed primary |
| Phase 3 | NCT02193022 | Jul 2014 → Jun 2019 | cutaneous leishmaniasis | International Centre for Diarrhoeal Disease Research, Bangladesh | Completed | No outcome recorded |
| Phase 3 | NCT02011958 | Jul 2014 → Aug 2016 | visceral leishmaniasis | Drugs for Neglected Diseases | Completed | No outcome recorded |
| Phase 3 | NCT01122771 | May 2010 → Mar 2014 | visceral leishmaniasis | Drugs for Neglected Diseases | Completed | Met primary |
| Phase 3 | NCT00696969 | Jun 2008 → Jan 2010 | visceral leishmaniasis | Drugs for Neglected Diseases | Completed | Met primary |
| Phase 3 | NCT00523965 | Sep 2007 → Aug 2009 | visceral leishmaniasis | Banaras Hindu University | Completed | No outcome recorded |
| Phase 3 | NCT00471705 | Jun 2006 → Aug 2008 | cutaneous leishmaniasis | Universidad de Antioquia | Completed | Missed primary |
| Phase 3 | NCT00351520 | May 2006 → Jun 2007 | cutaneous leishmaniasis | Tehran University of Medical Sciences | Completed | No outcome recorded |
| Phase 42 trials | ||||||
| Phase 4 | NCT01975051 | Jan 2013 → Sep 2014 | cutaneous leishmaniasis | International Centre for Diarrhoeal Disease Research, Bangladesh | Completed | No outcome recorded |
| Phase 4 | NCT01462500 | Oct 2011 → May 2014 | cutaneous leishmaniasis | International Center for Training and Medical Research | Completed | No outcome recorded |
| Comparator or background therapy6 trials | ||||||
| Phase 0 | NCT06251739 Comparator | Oct 2023 → Jan 2024 | cutaneous leishmaniasis | International Centre for Diarrhoeal Disease Research, Bangladesh | Completed | No outcome recorded |
| Phase 2 | NCT06997159 Comparator | Jun 2026 → Apr 2028 expected | cutaneous leishmaniasis | Drugs for Neglected Diseases | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT01380314 Background | Mar 2008 → Mar 2010 | cutaneous leishmaniasis | Foundation Fader | Completed | No outcome recorded |
| Phase 3 | NCT07463040 Comparator | Jan 2027 → Mar 2029 expected | cutaneous leishmaniasis | Institute of Tropical Medicine, Belgium | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT04799236 Comparator | Apr 2021 → Apr 2024 | mucocutaneous leishmaniasis | Fundacion Nacional de Dermatologia | Unknown | No outcome recorded |
| Phase 3 | NCT00487253 Comparator | Jul 2007 → Feb 2010 | cutaneous leishmaniasis | International Center for Training and Medical Research | Unknown | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2026-07-29 | Drugs for Neglected Diseases | Results WHO recommends new, better and safer treatment regimens for leishmaniasis dndi.org ↗
The MF+PM regimen demonstrated over 90% effectiveness and reduces treatment from 34 painful injections over 17 days to 14 injections over 14 days – 20 fewer injections – with one injection per day alongside oral MF. |
| 2024-07-17 | Drugs for Neglected Diseases | Results Clinical trial in Eastern Africa finds a shorter, safer, and more effective treatment for PKDL, a stigmatizing skin disease dndi.org ↗
At the 12-month follow-up visit, 98% of the participants had achieved a complete cure, including those with moderate and mild PKDL. |
| 2022-09-29 | Drugs for Neglected Diseases | Results Better, shorter combination treatment for people with visceral leishmaniasis shown to be effective in Eastern Africa dndi.org ↗
A shorter, and less toxic treatment for people with visceral leishmaniasis was shown to be effective thanks to a study conducted in Eastern Africa by the non-profit medical research organization Drugs for Neglected Diseases initiative (DNDi) and its partners. |
| 2022-06-08 | Drugs for Neglected Diseases | Results New WHO treatment guidelines offer hope for people co-infected with visceral leishmaniasis and HIV dndi.org ↗
In Ethiopia, the new treatment strategy was shown to have an 88% efficacy rate at the end of therapy (after 58 days), whereas the efficacy of the current standard treatment was 55% in the trial. |
| 2019-01-16 | Drugs for Neglected Diseases | Results Combination therapy found to be more effective in treating patients with leishmaniasis and HIV | DNDi dndi.org ↗ |
| 2014-10-14 | Drugs for Neglected Diseases | Results Study Results Support WHO Recommendations for Safe and Effective Kala-azar Treatments in Bangladesh | DNDi dndi.org ↗
The combination treatments (AmBisome® + miltefosine; AmBisome® + paromomycin; paromomycin + miltefosine) were non-inferior to AmBisome® monotherapy: the combinations of AmBisome® + paromomycin and paromomycin + miltefosine demonstrated excellent cure rates of above 98%, thus comparable with AmBisome® monotherapy. |
| 2014-03-19 | Knight Therapeutics (USA) Inc | Regulatory Knight Therapeutics Inc. Announces FDA Approval for Impavido® (Miltefosine) for the Treatment of Visceral, Mucosal and Cutaneous Leishmaniasis knighttx.com ↗
Knight Therapeutics Inc. (“Knight”) (TSX VENTURE:GUD) today announced that the U.S. Food and Drug Administration (FDA) has approved Impavido® (Miltefosine) for patients with Visceral, Mucosal and Cutaneous Leishmaniasis. |
| 2011-01-24 | Drugs for Neglected Diseases | Results Study shows the safety and efficacy of combination treatment for visceral leishmaniasis in India | DNDi dndi.org ↗
A phase III clinical trial study published online in The Lancet shows that three short-course combination treatments for visceral leishmaniasis in India are efficacious and safe. |
All press releases naming this drug 11 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 46 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Miltefosine | “Miltefosine is drug and has recently been shown to be effective in the treatment cutaneous leishmaniasis in Colombia.” NCT00351520 ↗42“Doxycycline, Paramomycin, Clarithromycin, Clindamycin, Dapsone, Miltefosine” NCT02366884 ↗ “Tablet Miltefosine 100 mg in two devided doses for 12 weeks” NCT01975051 ↗ |
| Known as | D-18506 | ChEMBL registry synonym — accepted as the source's own label CHEMBL125 ↗ |
| Known as | hexadecylphosphocholine | “Miltefosine (hexadecylphosphocholine) is an oral drug which has proven to be effective for the treatment of Visceral leishmaniasis (VL) in the Indian sub-continent.” NCT02687971 ↗2“Activities of hexadecylphosphocholine (miltefosine), AmBisome, and sodium stibogluconate (Pentostam) against Leishmania donovani in immunodeficient scid mice.” PMID 11353640 ↗ Jun 2001 “Activities of Hexadecylphosphocholine (Miltefosine), AmBisome, and Sodium Stibogluconate (Pentostam) against Leishmania donovani in Immunodeficient scid Mice.” NCT00351520 ↗ |
| Known as | Impavido | “Participants received miltefosine (Impavido) at a nominal dose of 2.5 mg/kg/day for 28 days.” PMID 27956421 ↗ Feb 20172“Miltefosine capsules (Impavido®) 2.5 mg/kg daily PO for 28 days” NCT04268524 ↗ “Miltefosine 50 MG Oral Capsule [Impavido]” NCT06997159 ↗ |
| Known as | Miltefosina | ChEMBL registry synonym — accepted as the source's own label CHEMBL125 ↗ |
| Known as | Miltefosine cap 10mg and 50mg, Impavido (Zentaris) | “Miltefosine cap 10mg and 50mg, Impavido® (Zentaris)” NCT00487253 ↗ |
| Known as | Miltex | ChEMBL registry synonym — accepted as the source's own label CHEMBL125 ↗ |
| Known as | NSC-605583 | ChEMBL registry synonym — accepted as the source's own label CHEMBL125 ↗ |
| Known as | NSC-758968 | ChEMBL registry synonym — accepted as the source's own label CHEMBL125 ↗ |
| Modality | Small molecule | “Miltefosine, is a phosphocholine analogue that affects cell-signaling pathways and membrane synthesis 5.” NCT00351520 ↗ |
| Route | Oral | “Miltefosine is an oral agent” NCT00233545 ↗ |