RepurpOS

Pulmonary Arterial Hypertension (PAH)

Disease intelligence for Pulmonary Arterial Hypertension: 18 alterations and 421 ranked therapeutics from curated public databases.

US DALYs3.13MWHO GHE 2021 · all ages
Global DALYs28.29MWHO GHE 2021 · worldwide
US mortality109.5K deaths/yrWHO GHE 2021
Global mortality1.02M deaths/yrWHO GHE 2021 · worldwide
NIH funding$377MFY2025 · RCDC estimate
Funding levelUnderfunded
Spontaneous remissionWithout treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year…
Best-intervention remissionTriple upfront combination therapy (ambrisentan + tadalafil + selexipag): 41% reduction in clinical failure events…
Gap sizeLarge — most patients still receive sequential monotherapy or dual therapy rather than upfront triple therapy
Primary barrierpractice/access
Full remission profile →

Disease overview

Alterations
18
Molecular (A)
5
Clinical (B–E)
13
Trial outcome measures (T)
0
Direct drugs
118
Via biomarker
304
Natural agents
0
Natural products
77
Merged ranked
421
With trial/literature evidence
25

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Examine.com, disease_db_100 · Pipeline phase 7 · Updated 2026-10-15

Remission & chronicity

Spontaneous remission
Without treatment, median survival from diagnosis 2.8 years (pre-modern-therapy era); with current therapy 5-year survival ~50-60%; spontaneous remission essentially unknown
Best-intervention remission
Triple upfront combination therapy (ambrisentan + tadalafil + selexipag): 41% reduction in clinical failure events (TRITON trial); macitentan + tadalafil initial combination: 41% risk reduction (AMBITION); lung transplantation: 50-60% 5-year survival
Drug-free remission
—
Relapse after remission
—
Chronicity
—
Gap size
Large — most patients still receive sequential monotherapy or dual therapy rather than upfront triple therapy
Primary barrier
practice/access
Barrier detail: Upfront combination therapy evidence is robust but drug costs (selexipag alone ~$150K/year) limit access; PAH is often missed or delayed in diagnosis (mean 2-year diagnostic delay); most patients are not treated at PAH expert centres where combination protocols are standard

Last SoC change: 2024 — sotatercept (Winrevair, activin signalling inhibitor) FDA approved — first new mechanism in 15 years; 2017 selexipag approved; upfront dual combination established 2015 (AMBITION); prostacyclin pathway drugs since 1995 (epoprostenol)

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

Identifiers: MONDO: MONDO:0015924 · ORPHA: ORPHA:182090

Alterations

18 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.

NameTypeSubtypeDirection FrequencyEvidenceSourcesLinks
ACVRL1
activin A receptor like type 1
Molecular Gene — — OT score 0.68 Open Targets
ABCC8
ATP binding cassette subfamily C member 8
Molecular Gene — — OT score 0.44 Open Targets
ABL1
ABL proto-oncogene 1, non-receptor tyrosine kinase
Molecular Gene — — OT score 0.40 Open Targets
ADORA1
adenosine A1 receptor
Molecular Gene — — OT score 0.21 Open Targets
ACE2
angiotensin converting enzyme 2
Molecular Gene — — OT score 0.12 Open Targets
Abnormal cardiovascular system physiology Pathology Pathological finding — frequent HPO annotation HPO, Monarch
Chest pain Pathology Pathological finding — occasional HPO annotation HPO, Monarch
Dyspnea Pathology Pathological finding — frequent HPO annotation HPO, Monarch
Elevated jugular venous pressure Lab / Clinical Lab value — occasional HPO annotation HPO, Monarch
Fatigue Functional Functional test — occasional HPO annotation HPO, Monarch
Heart murmur Pathology Pathological finding — occasional HPO annotation HPO, Monarch
Hepatomegaly Pathology Pathological finding — occasional HPO annotation HPO, Monarch
Palpitations Pathology Pathological finding — occasional HPO annotation HPO, Monarch
Pedal edema Pathology Pathological finding — — HPO annotation HPO, Monarch
Pulmonary arterial hypertension Pathology Pathological finding — very_frequent HPO annotation HPO, Monarch
Right ventricular dilatation Pathology Pathological finding — occasional HPO annotation HPO, Monarch
Syncope Pathology Pathological finding — occasional HPO annotation HPO, Monarch
Tricuspid regurgitation Pathology Pathological finding — occasional HPO annotation HPO, Monarch

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.

DrugTypePhaseEvidence ScorePrice (UK / US)Trials & literatureSourcesLinks
SIROLIMUS Small molecule Approved Strong 100 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
1 trials
View evidence

Registry trials

  • NCT02587325 — Phase 1/1b Study With Nab-sirolimus for Patients With Severe Pulmonary Arterial Hypertension (COMPLETED) PHASE1

Open Targets, ChEMBL
ACETAZOLAMIDE Repurposing Small molecule Approved Strong 95 UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs — DGIdb
ACETAZOLAMIDE Repurposing Small molecule Approved Strong 95 UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs — ChEMBL
ACETOHEXAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ACYCLOVIR Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ADENOSINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ADENOSINE TRIPHOSPHATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ALBUTEROL SULFATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ALFUZOSIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ALFUZOSIN HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AMINOPHYLLINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AMIODARONE HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
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
ARFORMOTEROL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ARFORMOTEROL TARTRATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASCIMINIB Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASCIMINIB HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASCORBIC ACID Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASENAPINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASPIRIN Repurposing Small molecule Approved Strong 95 UK Very low cost · US Very low cost — DGIdb
ATENOLOL Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs — DGIdb
AXITINIB Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AZACITIDINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BENZPHETAMINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BETAXOLOL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BETHANIDINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BICALUTAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL
BITOLTEROL MESYLATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BLINATUMOMAB Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BOSUTINIB Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BOSUTINIB MONOHYDRATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BUSULFAN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CABERGOLINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CABOZANTINIB S-MALATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CAFFEINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CAFFEINE CITRATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CARBACHOL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL
CARTEOLOL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CARTEOLOL HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence
DGIdb
CARVEDILOL Repurposing Small molecule Approved Strong 95 UK £2–£5 / 28 tabs · US $5–$10 / 30 tabs
7 trials 1 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT01723371 — Beta Blockers for Treatment of Pulmonary Arterial Hypertension in Children (WITHDRAWN) PHASE1, PHASE2
  • NCT02120339 — Carvedilol PAH A Pilot Study of Efficacy and Safety (TERMINATED) PHASE1
  • NCT01568645 — Imatinib and Carvedilol for High Blood Pressure in the Lungs in Adults With Sickle Cell Disease (WITHDRAWN) PHASE1
  • NCT00240656 — Spironolactone Combined With Captopril and Carvedilol for the Treatment of Pulmonary Arterial Hypertension (COMPLETED) PHASE1
  • NCT02507011 — Beta-blockers in Pulmonary Arterial Hypertension (TERMINATED) PHASE2
  • NCT01586156 — PAHTCH Pulmonary Arterial Hypertension Treatment With Carvedilol for Heart Failure (Carvedilol) (COMPLETED) NA
  • NCT00964678 — Pilot Study of the Safety and Efficacy of Carvedilol in Pulmonary Arterial Hypertension (COMPLETED) PHASE1, PHASE2

Published literature

DGIdb
CARVEDILOL PHOSPHATE Repurposing Small molecule Approved Strong 95 UK £2–£5 / 28 tabs · US $5–$10 / 30 tabs
No indexed hits
View evidence
DGIdb
CELECOXIB Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CELIPROLOL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CHLORPROPAMIDE 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
CLOMIPRAMINE 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
CLOTRIMAZOLE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CLOZAPINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
DrugTypePhaseEvidence ScorePrice (UK / US)SourcesLinks
ALBUTEROL Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
AV-101 Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
BARDOXOLONE METHYL Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
BERAPROST Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
EPOPROSTENOL Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
ESUBERAPROST Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
ESUBERAPROST SODIUM Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
IMATINIB Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
IMATINIB MESYLATE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
IPRATROPIUM BROMIDE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
MELATONIN Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
MILRINONE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
RALINEPAG Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
RANOLAZINE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
ROSUVASTATIN Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
SERALUTINIB Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
SILDENAFIL Small molecule Phase III Moderate 80 UK £2–£8 / 4–8 tabs (generic) · US $5–$15 / 10-30 tabs Open Targets
SITAXENTAN SODIUM Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
TREPROSTINIL DIOLAMINE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
TREPROSTINIL PALMITIL Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
TREPROSTINIL SODIUM Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
VARDENAFIL Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
2-METHOXYESTRADIOL Small molecule Phase II Emerging 60 Research / experimental compound — pricing not applicable for routine clinical use Open Targets
ANASTROZOLE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
APABETALONE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
ASPIRIN Small molecule Phase II Emerging 60 UK Very low cost · US Very low cost Open Targets
ATORVASTATIN Small molecule Phase II Emerging 60 UK £1–£4 / 28 tabs · US $5–$12 / 30–90 tabs Open Targets
BENZBROMARONE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
BQ-123 Biologic Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
CARVEDILOL Small molecule Phase II Emerging 60 UK £2–£5 / 28 tabs · US $5–$10 / 30 tabs Open Targets
CICLETANINE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
CXA-10 Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
DAPAGLIFLOZIN Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
EMPAGLIFLOZIN Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
ENRASENTAN Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
FAMOTIDINE Small molecule Phase II Emerging 60 UK £2–£4 / 28–60 tabs · US $5–$10 / 30–60 tabs (Cost Plus) Open Targets
FLUOXETINE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
FRESPACIGUAT Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
FULVESTRANT Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
GLUTAMINE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) Open Targets
DrugTypePhaseEvidence ScorePrice (UK / US)Trials & literatureSourcesLinks
ACETAZOLAMIDE Repurposing via AQP1 Small molecule Approved Strong 95 UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs — DGIdb
ACETAZOLAMIDE Repurposing via ABCC8 Small molecule Approved Strong 95 UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs — ChEMBL
ACETOHEXAMIDE Repurposing via ABCC8 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ACYCLOVIR Repurposing via ABL1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ADENOSINE Repurposing via ADORA1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ADENOSINE TRIPHOSPHATE Repurposing via ABL1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ALBUTEROL SULFATE Repurposing via ADRB2 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ALFUZOSIN Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ALFUZOSIN HYDROCHLORIDE Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AMINOPHYLLINE Repurposing via ADORA1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AMIODARONE HYDROCHLORIDE Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AMITRIPTYLINE HYDROCHLORIDE Repurposing via ADRA1A Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $4–$10 / 30 tabs — DGIdb
AMPHETAMINE Repurposing via ABCC8 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL
ARFORMOTEROL Repurposing via ADRB2 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ARFORMOTEROL TARTRATE Repurposing via ADRB2 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASCIMINIB Repurposing via BCR Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASCIMINIB HYDROCHLORIDE Repurposing via BCR Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASCORBIC ACID Repurposing via AQP1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASENAPINE Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
ASPIRIN Repurposing via ADORA1 Small molecule Approved Strong 95 UK Very low cost · US Very low cost — DGIdb
ATENOLOL Repurposing via ADRB2 Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs — DGIdb
AXITINIB Repurposing via ABL1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
AZACITIDINE Repurposing via ABL1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BENZPHETAMINE Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BETAXOLOL Repurposing via ADRB2 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BETHANIDINE Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BICALUTAMIDE Repurposing via ABCC8 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL
BITOLTEROL MESYLATE Repurposing via ADRB2 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BLINATUMOMAB Repurposing via BCR Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BOSUTINIB Repurposing via BCR Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BOSUTINIB MONOHYDRATE Repurposing via BCR Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
BUSULFAN Repurposing via BCR Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CABERGOLINE Repurposing via ADRA1A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CABOZANTINIB S-MALATE Repurposing via ABL1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CAFFEINE Repurposing via ADORA1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CAFFEINE CITRATE Repurposing via ADORA1 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CARBACHOL Repurposing via ABCC8 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL
CARTEOLOL Repurposing via ADRB2 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb
CARTEOLOL HYDROCHLORIDE Repurposing via ADRB3 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence
DGIdb
CARVEDILOL Repurposing via ADRB3 Small molecule Approved Strong 95 UK £2–£5 / 28 tabs · US $5–$10 / 30 tabs
7 trials 1 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT01723371 — Beta Blockers for Treatment of Pulmonary Arterial Hypertension in Children (WITHDRAWN) PHASE1, PHASE2
  • NCT02120339 — Carvedilol PAH A Pilot Study of Efficacy and Safety (TERMINATED) PHASE1
  • NCT01568645 — Imatinib and Carvedilol for High Blood Pressure in the Lungs in Adults With Sickle Cell Disease (WITHDRAWN) PHASE1
  • NCT00240656 — Spironolactone Combined With Captopril and Carvedilol for the Treatment of Pulmonary Arterial Hypertension (COMPLETED) PHASE1
  • NCT02507011 — Beta-blockers in Pulmonary Arterial Hypertension (TERMINATED) PHASE2
  • NCT01586156 — PAHTCH Pulmonary Arterial Hypertension Treatment With Carvedilol for Heart Failure (Carvedilol) (COMPLETED) NA
  • NCT00964678 — Pilot Study of the Safety and Efficacy of Carvedilol in Pulmonary Arterial Hypertension (COMPLETED) PHASE1, PHASE2

Published literature

DGIdb

Natural Products

77 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

DrugTypePhaseEvidence ScorePrice (UK / US)Trials & literatureSourcesLinks
Vitamin C Natural product Nutraceutical Phase I Emerging 10 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
2 trials
View evidence

Registry trials

  • NCT02966665 — : Vascular Function in Health and Disease (RECRUITING) PHASE1
  • NCT03451266 — Effects of Vitamin C Administration on Extravascular Lung Water in Patients With Severe Features of Preeclampsia (COMPLETED) PHASE1

Garlic Natural product Food compound Preclinical Emerging 10 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
3 pubs
View evidence
Omega-3 Fatty Acids Natural product Nutraceutical Phase II Emerging 10 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
2 trials
View evidence

Registry trials

  • NCT04031508 — Effect of a Parenteral Emulsion With Omega3 on Neonates With PPHN and CDH (ENROLLING_BY_INVITATION) PHASE2
  • NCT02966899 — Reversal of Right Ventricular Steatosis in Pulmonary Hypertension (WITHDRAWN) NA

Folate Natural product Nutraceutical Phase III Emerging 10 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
4 trials
View evidence

Registry trials

  • NCT07353827 — FOLFOX-HAIC Combined With Donafenib and Pucotenlimab as First-Line Treatment for Unresectable Intrahepatic Cholangiocarcinoma (NOT_YET_RECRUITING) PHASE2
  • NCT06648525 — Adebrelimab Combined with Irinotecan Liposomes, 5-FU, CF ± Lenvatinib As First-line Treatment for Advanced ICC (NOT_YET_RECRUITING) PHASE2
  • NCT03269110 — Folic Acid Clinical Trial: Follow up of Children (FACT 4 Child) (COMPLETED)
  • NCT07328919 — Efficacy and Safety of TT-00420 (Tinengotinib) Tablets Versus Chemotherapy in Patients With Advanced Intrahepatic Cholangiocarcinoma Harboring FGFR2 Fusions/Rearrangements or Mutations (NOT_YET_RECRUITING) PHASE3

N-Acetylcysteine Natural product Amino acid Preclinical Emerging 10 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
1 trials
View evidence

Registry trials

  • NCT04081012 — N-acetyl Cysteine in Post-reperfusion Pulmonary Injury in Chronic Thromboembolic Pulmonary Hypertension. (UNKNOWN) NA

L-Arginine Natural product Amino acid Approved Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
8 trials
View evidence

Registry trials

  • NCT01317134 — Endothelial Function in Patients With Pulmonary Arterial Hypertension (COMPLETED) NA
  • NCT00000623 — Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN) (COMPLETED)
  • NCT00327080 — Sildenafil to Treat HIV-Associated Pulmonary Hypertension (TERMINATED) PHASE1
  • NCT02966665 — : Vascular Function in Health and Disease (RECRUITING) PHASE1
  • NCT03649932 — Enteral L Citrulline Supplementation in Preterm Infants - Safety, Efficacy and Dosing (COMPLETED) PHASE1
  • NCT05439460 — Management of Acute Pulmonary Hypertensive Crisis in Children With Known Pulmonary Arterial Hypertension (COMPLETED) PHASE4
  • NCT03402191 — L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension (UNKNOWN) PHASE4
  • NCT01142219 — L-Arginine and Sickle Cell Disease (COMPLETED) PHASE3

Resveratrol Natural product Food compound Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
19 pubs
View evidence

Published literature

Saffron Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
No indexed hits
View evidence
L-Citrulline Natural product Amino acid Phase III Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
5 trials 1 Cochrane 1 Trial pubs
View evidence

Registry trials

  • NCT05636397 — Safety and PK-PD Study of Oral L-CIT in Preterm Infants With BPD±PH and NEC (RECRUITING) NA
  • NCT00335244 — Intravenous L-Citrulline to Treat Children Undergoing Heart Bypass Surgery (COMPLETED) PHASE3
  • NCT03542812 — L-citrulline and Pulmonary Hypertension Associated With Bronchopulmonary Dysplasia (TERMINATED) EARLY_PHASE1
  • NCT03649932 — Enteral L Citrulline Supplementation in Preterm Infants - Safety, Efficacy and Dosing (COMPLETED) PHASE1
  • NCT01683981 — Exercise Capacity and Quality of Life in Patients With PPH Receiving Short Term Oral L-Citrulline Malate (UNKNOWN) EARLY_PHASE1

Published literature

Grape Seed Extract Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
No indexed hits
View evidence
Melatonin Natural product Nutraceutical Phase III Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
2 trials 1 Trial pubs 1 RCT
View evidence

Registry trials

  • NCT07090720 — Melatonin Effects on the Left Ventricular Function in Neonates With Persistent Pulmonary Hypertension (COMPLETED) NA
  • NCT06685679 — Melatonin for Pulmonary Hypertension in Full Term Neonates (COMPLETED) PHASE3

Published literature

Sulforaphane Natural product Food compound Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
3 pubs
View evidence
Iron Natural product Nutraceutical Approved Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
8 trials 3 Trial pubs 2 RCT
View evidence

Registry trials

  • NCT01847352 — Iron Status and Hypoxic Pulmonary Vascular Responses (COMPLETED) NA
  • NCT04086537 — BMPR2 Mutations and Iron Metabolism in Pulmonary Arterial Hypertension (COMPLETED)
  • NCT00960921 — Study of the Effects of Iron on Lung Blood Pressure at High Altitude (WITHDRAWN) PHASE2
  • NCT00000623 — Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN) (COMPLETED)
  • NCT01623492 — Eisenmenger Quality Enhancement Research Initiative (COMPLETED)
  • NCT07359599 — The Impact of IV Iron on Exercise Capacity and Quality of Life in Pulmonary Hypertension (RECRUITING) PHASE4
  • NCT00952302 — Study of the Effects of Iron Levels on the Lungs at High Altitude (COMPLETED) NA
  • NCT02594917 — Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension (COMPLETED)

Published literature

Echinacea Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
No indexed hits
View evidence
Ashwagandha Natural product Ayurvedic Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
No indexed hits
View evidence
Bacopa Natural product Ayurvedic Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
No indexed hits
View evidence
Egcg Natural product Food compound Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
2 pubs
View evidence
Silymarin Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
1 pubs
View evidence
Turmeric Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
2 pubs
View evidence
Andrographis Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US)
No indexed hits
View evidence
Astragalus Natural product TCM herb Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Berberine Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Ginseng Natural product Botanical Preclinical Emerging 7 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Cannabidiol Natural product Food compound Preclinical Emerging 2 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Ginkgo Biloba Natural product Botanical Preclinical Emerging 2 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Astragaloside Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Ginsenosides Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Hydrogen: Molecular Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Dhea (Dehydroepiandrosterone) Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Sophora Flavescens Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Puerarin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Polyphenols Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Paeoniflorin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Wogonin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
18β-Glycyrrhetinic Acid Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Antioxidant Formulas Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Apple Polyphenols Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Caffeic Acid Phenethyl Ester Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Chrysin Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Genistein Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Formononetin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Magnolol Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Nobiletin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Plumbagin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Red Sage Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Tanshinone Iia Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Flavonoids Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Safflower Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
2-Methoxyestradiol Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Protandim Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Apigenin Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Blueberry Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Capsaicin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Celastrol Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Cyanidin 3-glucoside Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Salvia Miltiorrhiza Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
D-Ribose Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Hydrogen Water Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Isoliquiritigenin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Isoquercitrin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Kaempferol Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Luteolin Natural product Food compound Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Citrus Naringin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Poria Cocos Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Paeonol Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Phloretin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Beet Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Punicalagin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Xanthohumol Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Shikonin Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Ligustrazine Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Isoflavones Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Holy Basil Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Honokiol Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Urolithin a Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Betaine Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —
Baicalein Natural product Nutraceutical Preclinical Emerging 0 Varies — openprescribing.net (UK) / costplusdrugs.com or GoodRx (US) —

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.

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