Primary Sclerosing Cholangitis (PSC) — RepurpOS

Disease overview

Alterations

187

Molecular (A)

100

Clinical (B–E)

Direct drugs

Via biomarker

Natural agents

Natural products

Merged ranked

127

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 cholestatic disease; median time to liver failure or transplant 12-15 years; spontaneous remission essentially zero; biliary strictures progress in majority; cholangiocarcinoma risk 10-15% lifetime

Best-intervention remission

Liver transplantation: 5-year survival 80-85% (curative but recurrence in 20% post-transplant); ursodeoxycholic acid (UDCA): improvement in LFTs but no transplant-free survival benefit (Phase 3 — no benefit, possibly harm at high dose); no pharmacotherapy proven to alter the natural history

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Very large — no approved disease-modifying therapy exists; liver transplantation is the only definitive treatment

Primary barrier

biological

Barrier detail: PSC has resisted all pharmacotherapy attempts; UDCA widely prescribed despite no survival benefit (at high doses — possibly harmful); the field has 20+ failed Phase 2/3 trials; norUDCA, nor-UDCA, and several FXR agonists are in Phase 3; gut-liver axis and gut microbiome increasingly implicated (80% of PSC patients have concurrent IBD)

Last SoC change: 2023 — multiple Phase 3 trials underway but no approval; UDCA re-evaluated (Phase 3 Mayo trial showed no benefit); annual colon and biliary cancer surveillance as major guideline update; no approved pharmacotherapy to date

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

Identifiers: MONDO: MONDO:0018646 · ORPHA: ORPHA:59303

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 Primary Sclerosing Cholangitis may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 8, Phase 2: 26, Phase 1: 6

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

187

Actionable

Pipeline Compounds (Top 6)

Bezafibrate, Bifidobacterium Spp., Cilofexor, Elafibranor, Norucholic Acid, Simvastatin

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 187 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abdominal pain	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal biliary tract morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal large intestine physiology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormal tubulointerstitial morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Acholic stools	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Acute hepatic failure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Adenocarcinoma of the large intestine	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Alopecia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Amyloid deposition	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ascites	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Autoimmunity	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bile duct proliferation	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Biliary cirrhosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Celiac disease	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cholangiocarcinoma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cholelithiasis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cholestasis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cholestatic liver disease	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic hepatic failure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic kidney disease	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cirrhosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Congestive heart failure	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased circulating vitamin D concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Decreased circulating vitamin E concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Decreased circulating vitamin K concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Depression	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dilated superficial abdominal veins	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dry skin	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ductal bile plugs	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Elevated alkaline phosphatase of hepatic origin	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating hepatic transaminase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated gamma-glutamyltransferase level	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Enamel hypoplasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Epidermal acanthosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Fatigue	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Fever	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Generalized amyotrophy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hearing impairment	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatic bridging fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatic failure	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
Hepatomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatosplenomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypoalbuminemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypodontia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ichthyosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intellectual disability	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Intrahepatic biliary atresia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Jaundice	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Mild intellectual disability	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Nephropathy	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Oligodontia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Orthokeratosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Osteopenia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Osteoporosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Palmar telangiectasia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pancreatitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Parakeratosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pleural effusion	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Polyclonal elevation of IgM	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Portal fibrosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Portal hypertension	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Prolonged prothrombin time	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pruritus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Reduced circulating vitamin A concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Renal insufficiency	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Sclerosing cholangitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Sparse eyebrow	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Sparse eyelashes	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Sparse hair	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Spider hemangioma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Splenomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thick hair	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Thyroiditis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Type I diabetes mellitus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ulcerative colitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Vesicoureteral reflux	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Weight loss	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
ACETAZOLAMIDE	None	Gene-targeted
ADALIMUMAB		Gene-targeted
ALDESLEUKIN		Gene-targeted
AMPHETAMINE	None	Gene-targeted
BELATACEPT		Gene-targeted
BICALUTAMIDE	None	Gene-targeted
CARBACHOL	None	Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CISPLATIN		Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
DEXAMETHASONE		Gene-targeted
DIAZEPAM	None	Gene-targeted
DICHLORPHENAMIDE	None	Gene-targeted
DIETHYLSTILBESTROL	None	Gene-targeted
DOCETAXEL ANHYDROUS		Gene-targeted
EPIRUBICIN	None	Gene-targeted
ETHOXZOLAMIDE	None	Gene-targeted
FLUDARABINE		Gene-targeted
FLUMAZENIL	None	Gene-targeted
FLUOROURACIL		Gene-targeted
HYDROQUINONE		Gene-targeted
IMATINIB		Gene-targeted
IMIPRAMINE	None	Gene-targeted
INDAPAMIDE	None	Gene-targeted
INDOMETHACIN	None	Gene-targeted
ISOSORBIDE DINITRATE	None	Gene-targeted
MAFENIDE	None	Gene-targeted
MECAMYLAMINE	None	Gene-targeted
MEDROXYPROGESTERONE ACETATE		Gene-targeted
METHAZOLAMIDE	None	Gene-targeted
METHOXSALEN	None	Gene-targeted
METOPROLOL	None	Gene-targeted
MIANSERIN	None	Gene-targeted
NALIDIXIC ACID	None	Gene-targeted
NICOTINE	None	Gene-targeted
NORFLOXACIN	None	Gene-targeted
OFLOXACIN	None	Gene-targeted
PHENYTOIN	None	Gene-targeted
PRAZOSIN	None	Gene-targeted
PREDNISOLONE		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
SIROLIMUS	Small molecule	Approved	Strong	100	5 pubs View evidence CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing	Small molecule	Approved	Strong	95	1 Cochrane 1 Meta-analysis View evidence Published literature Cochrane review Extraintestinal manifestations of inflammatory bowel disease: epidemiology, diagnosis, and management (Annals of medicine, 2010) Meta-analysis Efficacy and safety of biologics in primary sclerosing cholangitis with inflammatory bowel disease: A systematic review and meta-analysis (Hepatology communications, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
ALDESLEUKIN Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT01988506 — Induction of Regulatory t Cells by Low Dose il2 in Autoimmune and Inflammatory Diseases (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
BELATACEPT Repurposing	Small molecule	Approved	Strong	95	1 pubs 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
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	2 Trial pubs 1 RCT View evidence Published literature Clinical trial publication Noncholesterol Sterols as Surrogate Markers in Patients with Severe Alcoholic Hepatitis (Lipids, 2018) Clinical trial publication The role of dominant stenoses in bacterial infections of bile ducts in primary sclerosing cholangitis (European journal of gastroenterology & hepatology, 2006) CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing	Small molecule	Approved	Strong	95	1 Trial pubs 1 RCT View evidence Published literature Clinical trial publication Pembrolizumab plus concurrent chemoradiotherapy versus placebo plus concurrent chemoradiotherapy in patients with locally advanced squamous cell carcinoma of the head and neck (KEYNOTE-412): a randomised, double-blind, phase 3 trial (The Lancet. Oncology, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	7 pubs 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
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUDARABINE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing	Small molecule	Approved	Strong	95	1 trials 4 Trial pubs 2 RCT View evidence Registry trials NCT01549795 — Liver Transplantation for Hilar Cholangiocarcinoma in Association With Neoadjuvant Radio- and Chemo-therapy (UNKNOWN) NA CT.gov search · Cochrane · PubMed	DGIdb	—
HYDROQUINONE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMATINIB Repurposing	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MAFENIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MEDROXYPROGESTERONE ACETATE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
METHAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHOXSALEN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
NALIDIXIC ACID Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
NICOTINE Repurposing	Small molecule	Approved	Strong	95	2 Trial pubs View evidence Published literature Clinical trial publication No beneficial effects of transdermal nicotine in patients with primary sclerosing cholangitis: results of a randomized double-blind placebo-controlled cross-over study (European journal of gastroenterology & hepatology, 2001) Clinical trial publication Oral nicotine in treatment of primary sclerosing cholangitis: a pilot study (Digestive diseases and sciences, 1999) CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
NORFLOXACIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
OFLOXACIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
PHENYTOIN Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
PRAZOSIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
PREDNISOLONE Repurposing	Small molecule	Approved	Strong	95	1 trials 1 Trial pubs 1 RCT View evidence Registry trials NCT02703194 — Leflunomide for Maintenance of Remission in IgG4 Related Disease (COMPLETED) PHASE4 Published literature Clinical trial publication Noncholesterol Sterols as Surrogate Markers in Patients with Severe Alcoholic Hepatitis (Lipids, 2018) CT.gov search · Cochrane · PubMed	DGIdb	—
RISPERIDONE Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
STREPTOZOCIN Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
SULBACTAM Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
TAZOBACTAM Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
TROGLITAZONE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BEZAFIBRATE	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
BIFIDOBACTERIUM SPP.	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets
CILOFEXOR	Small molecule	Phase III	Moderate	80	—	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Sources	Links
BEZAFIBRATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BIFIDOBACTERIUM SPP.	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CILOFEXOR	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ELAFIBRANOR	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
NORUCHOLIC ACID	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
SIMVASTATIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
VANCOMYCIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
VEDOLIZUMAB	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ALDAFERMIN	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALDESLEUKIN	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ARAMCHOL MEGLUMINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BERBERINE URSODEOXYCHOLATE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BEXOTEGRAST	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CENICRIVIROC	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CLADRIBINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CM-101	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
COLESEVELAM HYDROCHLORIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
HYDROXOCOBALAMIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
HYMECROMONE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
LARSUCOSTEROL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
MARALIXIBAT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
MARALIXIBAT CHLORIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
MITOMYCIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
OBETICHOLIC ACID	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
RITIVIXIBAT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SELADELPAR	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SIMTUZUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
SULFASALAZINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TAURURSODIOL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
THALIDOMIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TIMOLUMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
TRETINOIN	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
URSODIOL	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
VIDOFLUDIMUS	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AMOXICILLIN	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
BUDESONIDE	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
CURCUMIN	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
DOCONEXENT	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
ERLOTINIB	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets
FENOFIBRATE	Small molecule	Phase I	Emerging	35	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing via ATG16L1	Small molecule	Approved	Strong	95	1 Cochrane 1 Meta-analysis View evidence Published literature Cochrane review Extraintestinal manifestations of inflammatory bowel disease: epidemiology, diagnosis, and management (Annals of medicine, 2010) Meta-analysis Efficacy and safety of biologics in primary sclerosing cholangitis with inflammatory bowel disease: A systematic review and meta-analysis (Hepatology communications, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
ALDESLEUKIN Repurposing via CD40	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT01988506 — Induction of Regulatory t Cells by Low Dose il2 in Autoimmune and Inflammatory Diseases (COMPLETED) PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
AMPHETAMINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
BELATACEPT Repurposing via CD28	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CIPROFLOXACIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	2 Trial pubs 1 RCT View evidence Published literature Clinical trial publication Noncholesterol Sterols as Surrogate Markers in Patients with Severe Alcoholic Hepatitis (Lipids, 2018) Clinical trial publication The role of dominant stenoses in bacterial infections of bile ducts in primary sclerosing cholangitis (European journal of gastroenterology & hepatology, 2006) CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
CISPLATIN Repurposing via CCL2	Small molecule	Approved	Strong	95	1 Trial pubs 1 RCT View evidence Published literature Clinical trial publication Pembrolizumab plus concurrent chemoradiotherapy versus placebo plus concurrent chemoradiotherapy in patients with locally advanced squamous cell carcinoma of the head and neck (KEYNOTE-412): a randomised, double-blind, phase 3 trial (The Lancet. Oncology, 2024) CT.gov search · Cochrane · PubMed	DGIdb	—
CLOMIPRAMINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DEXAMETHASONE Repurposing via BCL2L11	Small molecule	Approved	Strong	95	7 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing via CCL2	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPIRUBICIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUDARABINE Repurposing via CD40	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
FLUMAZENIL Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing via CCL2	Small molecule	Approved	Strong	95	1 trials 4 Trial pubs 2 RCT View evidence Registry trials NCT01549795 — Liver Transplantation for Hilar Cholangiocarcinoma in Association With Neoadjuvant Radio- and Chemo-therapy (UNKNOWN) NA CT.gov search · Cochrane · PubMed	DGIdb	—
HYDROQUINONE Repurposing via CD40	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMATINIB Repurposing via BCL2L11	Small molecule	Approved	Strong	95	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MAFENIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MEDROXYPROGESTERONE ACETATE Repurposing via CD28	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
METHAZOLAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METHOXSALEN Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
NALIDIXIC ACID Repurposing via ADCY3	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
NICOTINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	2 Trial pubs View evidence Published literature Clinical trial publication No beneficial effects of transdermal nicotine in patients with primary sclerosing cholangitis: results of a randomized double-blind placebo-controlled cross-over study (European journal of gastroenterology & hepatology, 2001) Clinical trial publication Oral nicotine in treatment of primary sclerosing cholangitis: a pilot study (Digestive diseases and sciences, 1999) CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
NORFLOXACIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
OFLOXACIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
PHENYTOIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
PRAZOSIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
PREDNISOLONE Repurposing via CD40	Small molecule	Approved	Strong	95	1 trials 1 Trial pubs 1 RCT View evidence Registry trials NCT02703194 — Leflunomide for Maintenance of Remission in IgG4 Related Disease (COMPLETED) PHASE4 Published literature Clinical trial publication Noncholesterol Sterols as Surrogate Markers in Patients with Severe Alcoholic Hepatitis (Lipids, 2018) CT.gov search · Cochrane · PubMed	DGIdb	—

Natural Products

2 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
Berberine Natural product Clinical trial publication A Randomized, Dose-Finding, Proof-of-Concept Study of Berberine Ursodeo… Clinical trial publication Berberine Ursodeoxycholate for the Treatment of Primary Sclerosing Chol…	Botanical	Preclinical	Emerging	7	2 Trial pubs 2 RCT View evidence Published literature Clinical trial publication A Randomized, Dose-Finding, Proof-of-Concept Study of Berberine Ursodeoxycholate in Patients With Primary Sclerosing Cholangitis (The American journal of gastroenterology, 2022) Clinical trial publication Berberine Ursodeoxycholate for the Treatment of Primary Sclerosing Cholangitis: The Search for the Elusive Pharmacologic Holy Grail Will Need to Continue (The American journal of gastroenterology, 2022) CT.gov search · Cochrane · PubMed		PubChem
Hesperidin Natural product Human Study Benefit of glucosyl Hesperidin in patients with primary biliary cholang…	Food compound	Preclinical	Emerging	0	No indexed hits View evidence 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.