Alpha-1 Antitrypsin Deficiency (AATD) — RepurpOS

Disease overview

Alterations

141

Molecular (A)

100

Clinical (B–E)

Direct drugs

Via biomarker

167

Natural agents

Natural products

Merged ranked

173

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Seeds, Examine.com · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

Progressive lung and liver disease; ZZ genotype: FEV1 decline accelerated (80-100 mL/year vs 30 mL/year in non-smokers); liver cirrhosis progressive; no spontaneous resolution without augmentation therapy

Best-intervention remission

Alpha-1 augmentation therapy (IV human AAT): slows CT-measured emphysema progression by 34% (RAPID trial); smoking cessation: most important modifiable factor (reduces FEV1 decline to near-normal rate); fazirsiran (RNAi targeting hepatic AAT production): 83% reduction in Z-AAT liver accumulation

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Very large — only 5-10% of AATD individuals are diagnosed; augmentation therapy reduces CT progression but functional benefit debated; liver disease has no approved disease-modifying therapy

Primary barrier

access + practice (underdiagnosis)

Barrier detail: AATD is misdiagnosed as COPD or asthma for a mean of 5-7 years; spirometry for COPD should trigger AATD testing but this is rarely done; fazirsiran (RNAi) reduces hepatic Z-protein accumulation — Phase 3 underway for liver disease; augmentation therapy is IV every 4 weeks lifelong (~$100K/year)

Last SoC change: 2022 — Phase 3 ATIXA trial of fazirsiran ongoing; RAPID trial results (2015) established augmentation therapy; IV augmentation therapy approved 1987; no major change in lung AATD management for 35 years

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0013282

Right to Try Relevance Critical

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Alpha-1 Antitrypsin Deficiency may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 2, Phase 2: 2, Phase 1: 1

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

141

Actionable

Pipeline Compounds (Top 6)

Fazirsiran, Mycophenolate Mofetil, Alvelestat, Belcesiran, Sodium Nitrite

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

Showing 80 of 141 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Asthma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bronchiectasis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bronchitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cholestasis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic bronchitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic pulmonary obstruction	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cirrhosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cough	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cytoplasmic antineutrophil antibody positivity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased DLCO	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dyspnea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating hepatic transaminase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Emphysema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Failure to thrive in infancy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastric varix	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hemoptysis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatic fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatocellular carcinoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Increased sputum production	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intrahepatic inclusion bodies	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Jaundice	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Neonatal unconjugated hyperbilirubinemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Panacinar emphysema	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Panniculitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Portal hypertension	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged neonatal jaundice	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Reduced circulating alpha-1-antitrypsin concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Splenomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Wheezing	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
ABCA3 ATP binding cassette subfamily A member 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ABCB11 ATP binding cassette subfamily B member 11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ABCB4 ATP binding cassette subfamily B member 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ABCC12 ATP binding cassette subfamily C member 12	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ABCC2 ATP binding cassette subfamily C member 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ABCG5 ATP binding cassette subfamily G member 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ACHE acetylcholinesterase (Yt blood group)	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ACOX1 acyl-CoA oxidase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ADA adenosine deaminase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ADAMTS18 ADAM metallopeptidase with thrombospondin type 1 motif 18	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ADGRF5 adhesion G protein-coupled receptor F5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
AIRE autoimmune regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ATP7A ATPase copper transporting alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Adverse Events (AEs)	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
Alpha 1-antitrypsin levels assessed from epithelial lining fluid (ELF) samples	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
BCS1L BCS1 ubiquinol-cytochrome c reductase complex chaperone	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
C3 complement C3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CAV1 caveolin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CEBPA CCAAT enhancer binding protein alpha	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CES1 carboxylesterase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CES1P1 carboxylesterase 1 pseudogene 1	Molecular	Gene	—	—	Emerging	Open Targets	Ensembl Open Targets
CFTR CF transmembrane conductance regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CITED2 Cbp/p300 interacting transactivator with ED-rich tail 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CRB3 crumbs cell polarity complex component 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
CYS1 cystin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DHCR7 7-dehydrocholesterol reductase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DNAH11 dynein axonemal heavy chain 11	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DNAH5 dynein axonemal heavy chain 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DPP4 dipeptidyl peptidase 4	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ELANE elastase, neutrophil expressed	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Endothelial Microparticles	Lab / Clinical	Lab value	—	—	Emerging	ClinicalTrials.gov	—
FAH fumarylacetoacetate hydrolase	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FBN1 fibrillin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FENDRR FOXF1 adjacent non-coding developmental regulatory RNA	Molecular	Gene	—	—	Emerging	Open Targets	Ensembl Open Targets
FOXF1 forkhead box F1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FOXP3 forkhead box P3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FSTL1 follistatin like 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
FUT8 fucosyltransferase 8	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
G6PC1 glucose-6-phosphatase catalytic subunit 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
GBA1 glucosylceramidase beta 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HMGN5 high mobility group nucleosome binding domain 5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HNF1A HNF1 homeobox A	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HOXA5 homeobox A5	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HVCN1 hydrogen voltage gated channel 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IFT88 intraflagellar transport 88	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IMPDH1 inosine monophosphate dehydrogenase 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
IMPDH2 inosine monophosphate dehydrogenase 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
INVS inversin	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
KL klotho	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
LEP leptin	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
ABACAVIR		Gene-targeted
ACETAZOLAMIDE	None	Gene-targeted
AMBENONIUM		Gene-targeted
AMBENONIUM CHLORIDE	inhibitor	Gene-targeted
AMITRIPTYLINE HYDROCHLORIDE		Gene-targeted
AMPHETAMINE	None	Gene-targeted
APIGENIN	inhibitor	Gene-targeted
APIXABAN		Gene-targeted
ATAZANAVIR		Gene-targeted
BENZBROMARONE		Gene-targeted
BENZGALANTAMINE GLUCONATE	inhibitor	Gene-targeted
BETAMETHASONE		Gene-targeted
BICALUTAMIDE	None	Gene-targeted
CARBACHOL	None	Gene-targeted
CARBOPLATIN		Gene-targeted
CEFACLOR ANHYDROUS		Gene-targeted
CEFADROXIL ANHYDROUS		Gene-targeted
CEFAMANDOLE		Gene-targeted
CEFOXITIN SODIUM		Gene-targeted
CEFTRIAXONE		Gene-targeted
CEFUROXIME AXETIL		Gene-targeted
CEPHALEXIN ANHYDROUS		Gene-targeted
CEPHRADINE		Gene-targeted
CHENODEOXYCHOLIC ACID		Gene-targeted
CHOLIC ACID		Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CISPLATIN		Gene-targeted
CLADRIBINE		Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
CLOZAPINE		Gene-targeted
CYCLOPHOSPHAMIDE ANHYDROUS		Gene-targeted
CYCLOSPORINE		Gene-targeted
DAUNORUBICIN LIPOSOMAL		Gene-targeted
DEFERASIROX		Gene-targeted
DEMECARIUM BROMIDE		Gene-targeted
DIAZEPAM	None	Gene-targeted
DICHLORPHENAMIDE	None	Gene-targeted
DICLOFENAC SODIUM		Gene-targeted
DIETHYLSTILBESTROL	None	Gene-targeted
DIPYRIDAMOLE		Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABACAVIR Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMBENONIUM Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AMBENONIUM CHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMITRIPTYLINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
APIGENIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
APIXABAN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATAZANAVIR Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENZBROMARONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BENZGALANTAMINE GLUCONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFACLOR ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFADROXIL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFAMANDOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFOXITIN SODIUM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFTRIAXONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFUROXIME AXETIL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEPHALEXIN ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CEPHRADINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CHENODEOXYCHOLIC ACID Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CHOLIC ACID Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLADRIBINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOZAPINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOSPORINE Repurposing	Small molecule	Approved	Strong	95	2 Trial pubs View evidence Published literature Clinical trial publication Long-term outcome in 42 pediatric liver transplant patients with alpha 1-antitrypsin deficiency: a single-center experience (Clinical transplantation, 2011) Clinical trial publication Weaning of immunosuppression in long-term liver transplant recipients (Transplantation, 1995) CT.gov search · Cochrane · PubMed	DGIdb	—
DAUNORUBICIN LIPOSOMAL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEFERASIROX Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEMECARIUM BROMIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICLOFENAC SODIUM Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIPYRIDAMOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DISTIGMINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DONEPEZIL Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DONEPEZIL HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
DOXORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ECHOTHIOPHATE IODIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ECOTHIOPATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
EDROPHONIUM CATION Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EDROPHONIUM CHLORIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
EFAVIRENZ Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Sources	Links
FAZIRSIRAN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MYCOPHENOLATE MOFETIL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ALVELESTAT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BELCESIRAN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SODIUM NITRITE	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
.ALPHA.1-PROTEINASE INHIBITOR HUMAN	Biologic	Preclinical	Emerging	15	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ABACAVIR Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
ACETAZOLAMIDE Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
AMBENONIUM Repurposing via ACHE	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AMBENONIUM CHLORIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMITRIPTYLINE HYDROCHLORIDE Repurposing via ADA	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
APIGENIN Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	—
APIXABAN Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
ATAZANAVIR Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
BENZBROMARONE Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
BENZGALANTAMINE GLUCONATE Repurposing via ACHE	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing via ACHE	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing via ATP7A	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFACLOR ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFADROXIL ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFAMANDOLE Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFOXITIN SODIUM Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFTRIAXONE Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEFUROXIME AXETIL Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEPHALEXIN ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CEPHRADINE Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CHENODEOXYCHOLIC ACID Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	DGIdb	—
CHOLIC ACID Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via ATP7A	Small molecule	Approved	Strong	95	—	DGIdb	—
CLADRIBINE Repurposing via ADA	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOMIPRAMINE Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOZAPINE Repurposing via C3	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
CYCLOSPORINE Repurposing via ABCB4	Small molecule	Approved	Strong	95	2 Trial pubs View evidence Published literature Clinical trial publication Long-term outcome in 42 pediatric liver transplant patients with alpha 1-antitrypsin deficiency: a single-center experience (Clinical transplantation, 2011) Clinical trial publication Weaning of immunosuppression in long-term liver transplant recipients (Transplantation, 1995) CT.gov search · Cochrane · PubMed	DGIdb	—
DAUNORUBICIN LIPOSOMAL Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	DGIdb	—
DEFERASIROX Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
DEMECARIUM BROMIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICLOFENAC SODIUM Repurposing via ABCC2	Small molecule	Approved	Strong	95	—	DGIdb	—
DIETHYLSTILBESTROL Repurposing via ABCB11	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIPYRIDAMOLE Repurposing via ADA	Small molecule	Approved	Strong	95	—	DGIdb	—

Natural Products

1 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked). Only agents with direct clinical evidence are shown by default.

Reference lookups: Examine.com

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
Vitamin E Natural product Human Study Vitamin E is able to reduce frequency of liver dysfunction in infants w…	Nutraceutical	Preclinical	Emerging	10	1 trials View evidence Registry trials NCT03470857 — Oxidoreductive Balance and Lysosomal Activity in Cancer Patients. (COMPLETED) CT.gov search · Cochrane · PubMed		PubChem

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.