Primary Sclerosing Cholangitis (PSC)

Disease overview

Alterations

Molecular (A)

Clinical (B–E)

Trial outcome measures (T)

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 2026-10-15

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:447771

Alterations

0 total · Types A (molecular) through E (functional). Molecular rows are Open Targets gene–disease associations (evidence column = OT association score), not measured levels; phenotypes are HPO terms annotated directly to this disease (Monarch); type T rows are outcome measures used in completed ClinicalTrials.gov studies, not alterations of the disease.

No alterations in this category.

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
SIROLIMUS	Small molecule	Approved	Strong	100	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	5 pubs View evidence CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
BELATACEPT Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	1 pubs 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
CIPROFLOXACIN Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	7 pubs 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
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPIRUBICIN Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	ChEMBL	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
FLUDARABINE Repurposing	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
FLUMAZENIL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMATINIB Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN 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	ChEMBL	ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
MAFENIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
MEDROXYPROGESTERONE ACETATE 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	—
METHAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
METHOXSALEN Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing	Small molecule	Approved	Strong	95	UK £2–£4 / 28 tabs · US $4–$9 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
NALIDIXIC ACID 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	ChEMBL	ChEMBL Open Targets
NICOTINE 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 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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
OFLOXACIN 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	ChEMBL	ChEMBL Open Targets
PHENYTOIN Repurposing	Small molecule	Approved	Strong	95	UK £3–£8 / supply · US $6–$15 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
PRAZOSIN Repurposing	Small molecule	Approved	Strong	95	UK £2–£6 / 28–60 tabs · US $5–$12 / 30–60 tabs	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
PREDNISOLONE Repurposing	Small molecule	Approved	Strong	95	UK £2–£5 / 28 tabs · US $5–$10 / 30 tabs	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
STREPTOZOCIN 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	—
SULBACTAM 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	ChEMBL	ChEMBL Open Targets
TACROLIMUS ANHYDROUS Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
TAZOBACTAM 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	ChEMBL	ChEMBL Open Targets
TROGLITAZONE Repurposing	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
BEZAFIBRATE	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	Open Targets	ChEMBL Open Targets
BIFIDOBACTERIUM SPP.	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	Open Targets	ChEMBL Open Targets
CILOFEXOR	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Price (UK / US)	Sources	Links
BEZAFIBRATE	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
BIFIDOBACTERIUM SPP.	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
CILOFEXOR	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
ELAFIBRANOR	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
NORUCHOLIC ACID	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
SIMVASTATIN	Small molecule	Phase III	Moderate	80	UK £1–£3 / 28 tabs · US $4–$10 / 30–90 tabs	Open Targets	ChEMBL Open Targets
VANCOMYCIN	Small molecule	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
VEDOLIZUMAB	Biologic	Phase III	Moderate	80	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
ALDAFERMIN	Biologic	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
ALDESLEUKIN	Biologic	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
ARAMCHOL MEGLUMINE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
BERBERINE URSODEOXYCHOLATE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
BEXOTEGRAST	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
CENICRIVIROC	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
CLADRIBINE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
CM-101	Biologic	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
COLESEVELAM HYDROCHLORIDE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
HYDROXOCOBALAMIN	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
HYMECROMONE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
LARSUCOSTEROL	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
MARALIXIBAT	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
MARALIXIBAT CHLORIDE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
MITOMYCIN	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
OBETICHOLIC ACID	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
RITIVIXIBAT	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
SELADELPAR	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
SIMTUZUMAB	Biologic	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
SULFASALAZINE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
TAURURSODIOL	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
THALIDOMIDE	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
TIMOLUMAB	Biologic	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
TRETINOIN	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
URSODIOL	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
VIDOFLUDIMUS	Small molecule	Phase II	Emerging	60	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
AMOXICILLIN	Small molecule	Phase I	Emerging	35	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
BUDESONIDE	Small molecule	Phase I	Emerging	35	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
CURCUMIN	Small molecule	Phase I	Emerging	35	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
DOCONEXENT	Small molecule	Phase I	Emerging	35	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
ERLOTINIB	Small molecule	Phase I	Emerging	35	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	Open Targets	ChEMBL Open Targets
FENOFIBRATE	Small molecule	Phase I	Emerging	35	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
ACETAZOLAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing via ATG16L1	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
BELATACEPT Repurposing via CD28	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
BICALUTAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CARBACHOL Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
CIPROFLOXACIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DEXAMETHASONE Repurposing via BCL2L11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	7 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
DIAZEPAM Repurposing via ADCY3	Small molecule	Approved	Strong	95	UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
DOCETAXEL ANHYDROUS Repurposing via CCL2	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
EPIRUBICIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	—	ChEMBL	ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
FLUDARABINE Repurposing via CD40	Small molecule	Approved	Strong	95	Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
FLUMAZENIL Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
FLUOROURACIL Repurposing via CCL2	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMATINIB Repurposing via BCL2L11	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	1 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
IMIPRAMINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
INDAPAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
INDOMETHACIN Repurposing via ADCY3	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	ChEMBL	ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
MAFENIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
MECAMYLAMINE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
MEDROXYPROGESTERONE ACETATE Repurposing via CD28	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	—
METHAZOLAMIDE Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
METHOXSALEN Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
METOPROLOL Repurposing via ADCY3	Small molecule	Approved	Strong	95	UK £2–£4 / 28 tabs · US $4–$9 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
MIANSERIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	—	ChEMBL	ChEMBL Open Targets
NALIDIXIC ACID Repurposing via ADCY3	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	ChEMBL	ChEMBL Open Targets
NICOTINE Repurposing via ADCY3	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 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	Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
OFLOXACIN Repurposing via ADCY3	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	ChEMBL	ChEMBL Open Targets
PHENYTOIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	UK £3–£8 / supply · US $6–$15 / 30 tabs	—	ChEMBL	ChEMBL Open Targets
PRAZOSIN Repurposing via ADCY3	Small molecule	Approved	Strong	95	UK £2–£6 / 28–60 tabs · US $5–$12 / 30–60 tabs	No indexed hits View evidence CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
PREDNISOLONE Repurposing via CD40	Small molecule	Approved	Strong	95	UK £2–£5 / 28 tabs · US $5–$10 / 30 tabs	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	Price (UK / US)	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	Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)	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	Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)	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.

Generated by OSMF Disease Intelligence Pipeline · Schema 1.1.0

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