Alpha-1 Antitrypsin Deficiency (AATD)

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

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

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	Price (UK / US)	Trials & literature	Sources	Links
ABACAVIR Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
AMBENONIUM Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AMBENONIUM CHLORIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	ChEMBL Open Targets
AMITRIPTYLINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	UK £1–£3 / 28 tabs · US $4–$10 / 30 tabs	—	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
APIGENIN Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
APIXABAN Repurposing	Small molecule	Approved	Strong	95	UK £20–£50 / 28-60 tabs · US $30–$70 / 30-60 tabs	—	DGIdb	—
ATAZANAVIR Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
BENZBROMARONE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
BENZGALANTAMINE GLUCONATE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
CEFACLOR ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFADROXIL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFAMANDOLE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFOXITIN SODIUM Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFTRIAXONE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFUROXIME AXETIL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEPHALEXIN ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEPHRADINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CHENODEOXYCHOLIC ACID Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CHOLIC ACID Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
CLADRIBINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CLOZAPINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
CYCLOSPORINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DEFERASIROX Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DEMECARIUM BROMIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DICLOFENAC SODIUM Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DIPYRIDAMOLE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DISTIGMINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
DONEPEZIL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DONEPEZIL HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	ChEMBL Open Targets
DOXORUBICIN HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
ECHOTHIOPHATE IODIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
ECOTHIOPATE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
EDROPHONIUM CATION Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EDROPHONIUM CHLORIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	ChEMBL Open Targets
EFAVIRENZ Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—

Drug	Type	Phase	Evidence	Score	Price (UK / US)	Sources	Links
FAZIRSIRAN	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
MYCOPHENOLATE MOFETIL	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
ALVELESTAT	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
BELCESIRAN	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
SODIUM NITRITE	Small molecule	Phase I	Emerging	35	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
.ALPHA.1-PROTEINASE INHIBITOR HUMAN	Biologic	Preclinical	Emerging	15	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Price (UK / US)	Trials & literature	Sources	Links
ABACAVIR Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
ACETAZOLAMIDE Repurposing via ABCB11	Small molecule	Approved	Strong	95	UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
AMBENONIUM Repurposing via ACHE	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
AMBENONIUM CHLORIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	ChEMBL Open Targets
AMITRIPTYLINE HYDROCHLORIDE Repurposing via ADA	Small molecule	Approved	Strong	95	UK £1–£3 / 28 tabs · US $4–$10 / 30 tabs	—	DGIdb	—
AMPHETAMINE Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
APIGENIN Repurposing via ACHE	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
APIXABAN Repurposing via ABCC2	Small molecule	Approved	Strong	95	UK £20–£50 / 28-60 tabs · US $30–$70 / 30-60 tabs	—	DGIdb	—
ATAZANAVIR Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
BENZBROMARONE Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
BENZGALANTAMINE GLUCONATE Repurposing via ACHE	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE Repurposing via ACHE	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CARBOPLATIN Repurposing via ATP7A	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
CEFACLOR ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFADROXIL ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFAMANDOLE Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFOXITIN SODIUM Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFTRIAXONE Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEFUROXIME AXETIL Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEPHALEXIN ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CEPHRADINE Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CHENODEOXYCHOLIC ACID Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CHOLIC ACID Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CIPROFLOXACIN Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via ATP7A	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
CLADRIBINE Repurposing via ADA	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CLOMIPRAMINE Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CLOZAPINE Repurposing via C3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
CYCLOPHOSPHAMIDE ANHYDROUS Repurposing via ABCC2	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	DGIdb	—
CYCLOSPORINE Repurposing via ABCB4	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DEFERASIROX Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DEMECARIUM BROMIDE Repurposing via ACHE	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via ABCB11	Small molecule	Approved	Strong	95	UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DICLOFENAC SODIUM Repurposing via ABCC2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	DGIdb	—
DIETHYLSTILBESTROL Repurposing via ABCB11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DIPYRIDAMOLE Repurposing via ADA	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	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	Price (UK / US)	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	Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)	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.