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Ambisome + Miltefosine
Ambisome (liposomal amphotericin B) disrupts fungal cell membranes while miltefosine inhibits phosphatidylcholine synthesis, together providing synergistic antileishmanial and antifungal activity.
Ambisome (liposomal amphotericin B) disrupts fungal cell membranes while miltefosine inhibits phosphatidylcholine synthesis, together providing synergistic antileishmanial and antifungal activity. Used for Visceral leishmaniasis (kala-azar), Cutaneous leishmaniasis.
At a glance
| Generic name | Ambisome + Miltefosine |
|---|---|
| Sponsor | Drugs for Neglected Diseases |
| Drug class | Antifungal/antiparasitic combination |
| Target | Ergosterol (amphotericin B); phosphatidylcholine synthesis pathway (miltefosine) |
| Modality | Small molecule |
| Therapeutic area | Infectious Disease / Neglected Tropical Diseases |
| Phase | Phase 3 |
Mechanism of action
Amphotericin B binds to ergosterol in fungal and parasitic cell membranes, causing membrane disruption and cell death. Miltefosine is an alkylphospholipid that inhibits phosphatidylcholine synthesis and induces apoptosis in Leishmania parasites. The combination leverages two distinct mechanisms to enhance efficacy against visceral leishmaniasis and other neglected tropical diseases.
Approved indications
- Visceral leishmaniasis (kala-azar)
- Cutaneous leishmaniasis
Common side effects
- Nephrotoxicity
- Infusion-related reactions
- Gastrointestinal disturbances
- Electrolyte abnormalities
- Hepatotoxicity
Key clinical trials
- Study Comparing Several Drugs to Understand Which Work Against Cutaneous Leishmaniasis (CL) (PHASE3)
- Treatment of Mucosal Bolivian Leishmaniasis (PHASE3)
- Pilot Study: Oral Treatment of American Tegumentary Leishmaniasis (Cutaneous and Mucosal Forms) in the Elderly (PHASE2, PHASE3)
- Study of Cases of Cutaneous Leishmaniasis Treated With Miltefosine in French Guiana, Retrospective Study
- Clinical Evaluation of a New Form of Cancer Therapy (Atavistic Chemotherapy) Based on the Principles of Atavistic Metamorphosis (2011) (PHASE2)
- Short Course Regimens for Treatment of PKDL (Sudan) (PHASE2)
- Efficacy Trial of Ambisome Given Alone and Ambisome Given in Combination With Miltefosine for the Treatment of VL HIV Positive Ethiopian Patients. (PHASE3)
- Clinical Trial to Assess the Safety and Efficacy of Sodium Stibogluconate (SSG) and AmBisome® Combination, Miltefosine and AmBisome® and Miltefosine Alone for the Treatment Visceral Leishmaniasis in Eastern Africa (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ambisome + Miltefosine CI brief — competitive landscape report
- Ambisome + Miltefosine updates RSS · CI watch RSS
- Drugs for Neglected Diseases portfolio CI