Amphotericin B

Summary

Amphotericin B (CHEMBL267345) is an approved small-molecule antiprotozoal drug (ATC J02AA01); indicated across 31 conditions including cryptococcal meningitis and histoplasmosis; with CIViC clinical evidence for 1 variant-indication association (e.g. ATP7B Expression in ovarian cancer).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J02AA01 (+3 more)
• Indications: 31 conditions
• Clinical trials: 73
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 924.1 Da · C47H73NO17

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL267345
Name	Amphotericin B
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	5280965
ChEBI	CHEBI:2682
ATC	J02AA01, A01AB04, A07AA07, G01AA03
Molecular formula	C47H73NO17
Molecular weight	924.1
InChIKey	APKFDSVGJQXUKY-INPOYWNPSA-N

SMILES: C[C@H]1/C=C/C=C/C=C/C=C/C=C/C=C/C=C/[C@@H](C[C@H]2[C@@H]([C@H](C[C@](O2)(C[C@H](C[C@H]([C@@H](CC[C@H](C[C@H](CC(=O)O[C@H]([C@@H]([C@@H]1O)C)C)O)O)O)O)O)O)O)C(=O)O)O[C@H]3[C@H]([C@H]([C@@H]([C@H](O3)C)O)N)O

IUPAC name: (1R,3S,5R,6R,9R,11R,15S,16R,17R,18S,19E,21E,23E,25E,27E,29E,31E,33R,35S,36R,37S)-33-[(2R,3S,4S,5S,6R)-4-amino-3,5-dihydroxy-6-methyloxan-2-yl]oxy-1,3,5,6,9,11,17,37-octahydroxy-15,16,18-trimethyl-13-oxo-14,39-dioxabicyclo[33.3.1]nonatriaconta-19,21,23,25,27,29,31-heptaene-36-carboxylic acid

ChEBI definition: A macrolide antibiotic used to treat potentially life-threatening fungal infections.

Pharmacological roles (ChEBI): antiprotozoal drug, antiamoebic agent.

Other ChEBI roles (chemical / environmental): bacterial metabolite.

Also known as: Abelcet, liposomal amphotericin b, Ambil, Ambisome, Amfotericina b, Amophotericin b, Ampho-moronal, Amphotec, Amphotercin b, Amphotericin, Amphotericin b, Amphotericin b deoxycholate

Patent coverage: 25,053 distinct patent families (86,737 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 9 (assay-derived). Sample: Prelamin-A/C, 4’-phosphopantetheinyl transferase ffp, Sodium-dependent serotonin transporter, Prostaglandin G/H synthase 2, Sodium-dependent dopamine transporter, Cruzipain, Cytochrome P450 2C19, Prostaglandin G/H synthase 1, 3-hydroxyacyl-CoA dehydrogenase type-2.

Bioactivity

ChEMBL activities: 4 potent at pChembl ≥ 5 of 10 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
LMNA	7.1	Potency	79.4	nM	CHEMBL_ACT_3663703
Q63921	5.96	AC50	1100	nM	CHEMBL_ACT_25173907
PTGS2	5.33	AC50	4700	nM	CHEMBL_ACT_25165709
P25779	5.1	Potency	7943	nM	CHEMBL_ACT_3991247

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

9 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
Cryptococcal meningitis	4	MONDO:0005723	EFO:0007228
histoplasmosis	4	MONDO:0018312	EFO:0007310
Zygomycosis	4	MONDO:0019136	EFO:0007380
sporotrichosis	4	MONDO:0005968	EFO:0007494
aspergillosis	4	MONDO:0005657	EFO:0007157
cryptococcosis	4	MONDO:0005724	EFO:0007229
visceral leishmaniasis	4	MONDO:0005445	EFO:0005045
candidiasis	4	MONDO:0002026	MONDO:0002026
fungal infectious disease	4	MONDO:0002041	MONDO:0002041

21 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
leishmaniasis	3	MONDO:0011989	EFO:0005044
myelodysplastic syndrome	3	MONDO:0018881	EFO:0000198
leukemia	3	MONDO:0005059	EFO:0000565
lung disorder	3	MONDO:0005275	EFO:0003818
plasma cell myeloma	3	MONDO:0009693	EFO:0001378
myeloproliferative neoplasm	3	MONDO:0020076	EFO:0002428
bacterial vaginosis	3	MONDO:0005316	EFO:0003932
blastomycosis	3	MONDO:0005672	EFO:0007174
vulvovaginal candidiasis	3	MONDO:0006014	EFO:0007543
mucocutaneous leishmaniasis	3	MONDO:0005859	EFO:0007379
neoplasm	3	MONDO:0005070	MONDO:0004992
lymphoma	3	MONDO:0005062	EFO:0000574
pulmonary aspergilloma	3	MONDO:0000266	EFO:1001834
candidiasis, invasive	2	MONDO:0044067	EFO:1001283
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
allergic bronchopulmonary aspergillosis	2	MONDO:0015243	EFO:0007140
intracranial hypertension	2	MONDO:0006810	EFO:1000992
acute lymphoblastic leukemia	2	MONDO:0004967	EFO:0000220
Fuchs’ endothelial dystrophy	2	MONDO:0005321	Orphanet:98974
chronic mucocutaneous candidiasis	2	MONDO:0015279	Orphanet:1334
cutaneous leishmaniasis	1	MONDO:0005446	EFO:0005046

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 73.

Phase distribution

Phase	Trials
PHASE3	19
PHASE4	14
Not specified	14
PHASE2	13
PHASE1	7
PHASE1/PHASE2	3
PHASE2/PHASE3	2
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06178627	PHASE4	ENROLLING_BY_INVITATION	Amphotericin B for Non-HIV Cryptococcal Meningitis Patients
NCT00001107	PHASE4	COMPLETED	Comparison of Two Treatments to Prevent Invasive Fungal Infections in Patients Who Have Received Liver Transplants
NCT00161356	PHASE4	TERMINATED	Ambisome in Liver Transplant Patients
NCT00190463	PHASE4	TERMINATED	Comparison of 2 Antifungal Treatment (Empirical Versus Pre-Empirical) Strategies in Prolonged Neutropenia
NCT00334412	PHASE4	COMPLETED	COMBISTRAT: AmBisome® in Combination With Caspofungin for the Treatment of Invasive Aspergillosis
NCT00362544	PHASE4	COMPLETED	PROPHYSOME: Pilot Study on Safety of a Weekly Administration of 10mg/kg of AmBisome® in Antifungal Prophylaxis Treatment of Allogeneic Stem-cell Transplantation and Acute Leukaemia
NCT00670657	PHASE4	COMPLETED	CRITIC - Treatment of Candidemia and Invasive Candidiasis
NCT00986713	PHASE4	UNKNOWN	Value of Amphotericin B Inhalation for Prophylaxis of Invasive Pulmonary Aspergillosis After Renal Transplantation
NCT01032187	PHASE4	COMPLETED	Amphotericin B to Treat Visceral Leishmaniasis in Brazilian Children
NCT01310738	PHASE4	TERMINATED	Efficacy and Safety Study of Drugs for Treatment of Visceral Leishmaniasis in Brazil
NCT01566552	PHASE4	UNKNOWN	Single Dose Liposomal Amphotericin B for Visceral Leishmaniasis
NCT02283905	PHASE4	TERMINATED	Amphotericin-B and Voriconazole for Pulmonary Blastomycosis
NCT03311607	PHASE4	COMPLETED	Safety and Effectiveness of Short-course AmBisome in the Treatment of PKDL in Bangladesh
NCT06413056	PHASE4	COMPLETED	Micafungin Versus Amphotercine B in Treatment of Invasive Fungal Infection
NCT06447402	PHASE3	RECRUITING	A Trial to Compare Nebulized Amphotericin B and Nebulized Normal Saline as Maintenance in Patients With Chronic Pulmonary Aspergillosis
NCT00001017	PHASE3	COMPLETED	Comparison of Fluconazole and Amphotericin B in the Treatment of Brain Infections in Patients With AIDS
NCT00001646	PHASE3	COMPLETED	Voriconazole vs. Amphotericin B in the Treatment of Invasive Aspergillosis
NCT00002159	PHASE3	COMPLETED	A Randomized, Open, Comparative Multicenter Study of Initial Treatment With Intravenous Itraconazole Versus Amphotericin B Followed by Consolidation Treatment With Itraconazole Capsules in Patients With Blastomycosis or Histoplasmosis
NCT00002277	PHASE3	COMPLETED	A Comparison of Fluconazole and Amphotericin B in the Treatment of Fungal Infections
NCT00002742	PHASE3	COMPLETED	Antifungal Therapy for Fever and Neutropenia in Patients Receiving Treatment for Hematologic Cancer
NCT00003031	PHASE3	COMPLETED	Comparison of Voriconazole and Amphotericin B in Treating Patients With Aspergillosis
NCT00158730	PHASE3	COMPLETED	Study Safety/Efficacy of AmBisome Loading Dose Regimen Versus Standard AmBisome Regimen for Initial Treatment
NCT00177684	PHASE3	COMPLETED	Pharmacokinetic Profiles of Inhaled Lipid Complex Amphotericin B (Abelcet ®)
NCT00177710	PHASE3	COMPLETED	Pharmacokinetic Profile of Inhaled Liposomal Amphotericin B in Lung Transplant Recipients - Ambisome Study
NCT00216346	PHASE3	COMPLETED	Safety and Efficacy Study of Paromomycin to Treat Visceral Leishmaniasis
NCT00235651	PHASE3	TERMINATED	Abelcet Radiotagging Protocol: Inhaled Lipid Complex Abelcet® in Lung Transplant Recipients
NCT00361517	PHASE3	COMPLETED	To Determine Whether Galactomannan Test Will Help to Detect Fungal Infections Early and Hence Start Treatment Early
NCT00523965	PHASE3	COMPLETED	Combination Therapy in Indian Visceral Leishmaniasis
NCT00628719	PHASE3	COMPLETED	Single Infusion of Liposomal Amphotericin B in Indian Visceral Leishmaniasis
NCT00696969	PHASE3	COMPLETED	Safety and Efficacy Study to Evaluate Different Combination Treatment Regimens for Visceral Leishmaniasis
NCT00815516	PHASE3	TERMINATED	Study to Compare the Efficacy and Safety of Micafungin Versus Conventional Amphotericin B for the Treatment of Neonatal Candidiasis
NCT01857479	PHASE2/PHASE3	COMPLETED	A Randomized Controlled Trial of Inhaled Amphotericin B for Maintaining Remission in Allergic Bronchopulmonary Aspergillosis
NCT04018417	PHASE2/PHASE3	WITHDRAWN	Evaluation of Amphotericin B in Optisol-GS for Prevention of Post-Keratoplasty Fungal Infections.
NCT04140461	PHASE3	UNKNOWN	AmB Dose for Cryptococcal Meningitis
NCT05541107	PHASE3	UNKNOWN	Encochleated Oral Amphotericin for Cryptococcal Meningitis Trial 3
NCT00000776	PHASE2	COMPLETED	Dexamethasone in Cryptococcal Meningitis
NCT00001065	PHASE2	COMPLETED	A Study of Amphotericin B in the Treatment of Fungal Infections of the Mouth in HIV-Infected Patients Who Have Not Had Success With Fluconazole
NCT00012467	PHASE2	COMPLETED	Safety and Antifungal Activity of Recombinant Interferon-Gamma 1b (rIFN-Gamma 1b) Given With Standard Therapy in Patients With Cryptococcal Meningitis
NCT00082537	PHASE2	COMPLETED	MK0991 Versus Amphotericin B for Empirical Therapy in Febrile, Neutropenic Pediatric Patients (0991-044)
NCT00145249	PHASE2	COMPLETED	Amphotericin Alone or in Combination With Fluconazole for AIDS-Associated Meningitis
NCT00326157	PHASE2	COMPLETED	PROPHESSOR: AmBisome in Antifungal Primary Prophylaxis Treatment of High Risk Patients Undergoing Allogeneic Stem Cell Transplantation
NCT00847678	PHASE2	TERMINATED	Efficacy and Safety of Mycograb as Adjunctive Therapy for Cryptococcal Meningitis in Patients With AIDS
NCT00885703	PHASE1/PHASE2	COMPLETED	High-Dose Fluconazole for the Treatment of Cryptococcal Meningitis in HIV-Infected Individuals
NCT01437020	PHASE1/PHASE2	WITHDRAWN	SCH708980 With and Without AmBisome for Visceral Leishmaniasis
NCT01615809	PHASE2	COMPLETED	Nebulized Amphotericin B Lipid Complex in Invasive Pulmonary Aspergillosis in Paediatric Patients With Acute Leukaemia
NCT01945281	PHASE2	TERMINATED	Safety, Tolerability, and Efficacy of Caspofungin Versus Amphotericin B Deoxycholate in the Treatment of Invasive Candidiasis in Neonates and Infants (MK-0991-064)
NCT02629419	PHASE2	COMPLETED	CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis
NCT02656797	PHASE2	COMPLETED	Topical Liposomal Amphotericin B Gel Treatment for Cutaneous Leishmaniasis
NCT03112031	PHASE2	COMPLETED	Treatment With Tamoxifen in Cryptococcal Meningitis
NCT03399955	PHASE2	UNKNOWN	Short Course Regimens for Treatment of PKDL (Sudan)
NCT04031833	PHASE1/PHASE2	COMPLETED	Encochleated Oral Amphotericin for Cryptococcal Meningitis Trial (EnACT)
NCT00002316	PHASE1	COMPLETED	The Safety and Effectiveness of RMP-7 Plus Amphotericin B in Patients With HIV and Cryptococcal Meningitis
NCT01652859	PHASE1	COMPLETED	An Bioequivalence Study to Compare Two 2 mg/ml Liposomal Amphotericin B Injections in Healthy Subjects
NCT03636659	PHASE1	COMPLETED	Steady State Global Bioequivalence Study of Amphotericin B Liposome for Injection 50 mg/ Vial in Fed Condition
NCT04267497	PHASE1	COMPLETED	Nebulised Liposomal Amphotericin for Invasive Pulmonary Aspergillosis (NAIFI01 Study)
NCT04993222	PHASE1	COMPLETED	A Pilot Bioequivalence Study Between Amphotericin B Liposome for Injection and AmBisome® in Healthy Subjects
NCT05749380	PHASE1	COMPLETED	Pharmacokinetics and Safety of AmBisome and DKF-5122
NCT05913921	PHASE1	COMPLETED	Bioequivalence of Amphotericin B Liposome for Injection
NCT04072640	EARLY_PHASE1	UNKNOWN	Three Induction Treatments on Cryptococcal Meningitis
NCT06926569	Not specified	NOT_YET_RECRUITING	Single High-dose of Liposomal Amphotericin B in Combination With B/F/TAF for HIV/AIDS-associated Talaromycosis
NCT07191756	Not specified	ACTIVE_NOT_RECRUITING	Amphotericin B Versus Isavuconazole for Mucormycosis: A Comparative Efficacy and Safety Study
NCT07559032	Not specified	NOT_YET_RECRUITING	Amphotericin B for Breakthrough Fungal Infections After Triazole Prophylaxis in Hematological Patients
NCT00000639	Not specified	COMPLETED	A Randomized Double Blind Protocol Comparing Amphotericin B With Flucytosine to Amphotericin B Alone Followed by a Comparison of Fluconazole and Itraconazole in the Treatment of Acute Cryptococcal Meningitis
NCT00000708	Not specified	COMPLETED	Multi-center Comparison of Fluconazole (UK-49,858) and Amphotericin B as Treatment for Acute Cryptococcal Meningitis
NCT00002019	Not specified	COMPLETED	Safety and Efficacy of Amphotericin B Lipid Complex in the Treatment of Cryptococcal Meningitis in Patients With the Acquired Immunodeficiency Syndrome
NCT00002041	Not specified	COMPLETED	Evaluation of Amphotericin B in the Treatment of Biopsy Proven Candida Esophagitis in Immunocompromised Patients
NCT00002068	Not specified	COMPLETED	A Multicenter Comparison of Fluconazole (UK-49,858) and Amphotericin B as Treatment for Acute Cryptococcal Meningitis
NCT00002074	Not specified	COMPLETED	Multicenter Comparison of Fluconazole (UK-49,858) and Amphotericin B as Maintenance Treatment for the Prevention of Relapse of Cryptococcal Meningitis in Patients With Acquired Immunodeficiency Syndrome
NCT00002075	Not specified	COMPLETED	Multicenter Comparison of Fluconazole (UK-49,858) and Amphotericin B as Treatment for Acute Cryptococcal Meningitis
NCT00002305	Not specified	COMPLETED	A Comparison of Fluconazole and Amphotericin B in the Treatment of Cryptococcal Meningitis
NCT00491426	Not specified	COMPLETED	Antimicrobial PK in Infants With Suspected or Confirmed Infection
NCT02136030	Not specified	UNKNOWN	Liposomal Amphotericin B for the Treatment of Cryptococcal Meningitis
NCT04532463	Not specified	COMPLETED	Clinical Effectiveness and Safety of Amphotericin B With Flucytosine-Fluconazole Therapy for Cryptococcal Meningitis

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
ATP7B Expression	Ovarian Cancer	Sensitivity/Response	Amphotericin B	CIViC D	EID12305

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).