RepurpOS

Pulmonary Arterial Hypertension (PAH)

Disease intelligence for Pulmonary Arterial Hypertension: 183 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
183
Molecular (A)
100
Clinical (B–E)
83
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 2026-07-06

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:275777

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 Pulmonary Arterial Hypertension may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.
Investigational Agents
40
Phase 3: 22, Phase 2: 18
Most Advanced Phase
Phase 3
Highest dev. stage
Gene Targets
183
Actionable
Pipeline Compounds (Top 6)
Albuterol, Av-101, Bardoxolone Methyl, Beraprost, Epoprostenol, Esuberaprost
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 183 · Types A (molecular) through E (functional)

NameTypeSubtypeDirection FrequencyEvidenceSourcesLinks
Abnormal pulmonary interstitial morphology Pathology Pathological finding Moderate HPO, Monarch
Abnormal thrombosis Pathology Pathological finding Moderate HPO, Monarch
Abnormally loud pulmonic component of the second heart sound Pathology Pathological finding Moderate HPO, Monarch
Alveolar septal thickening Pathology Pathological finding Moderate HPO, Monarch
Angina pectoris Pathology Pathological finding Moderate HPO, Monarch
Antinuclear antibody positivity Pathology Pathological finding Moderate HPO, Monarch
Apnea Pathology Pathological finding Moderate HPO, Monarch
Arterial intimal fibrosis Pathology Pathological finding Moderate HPO, Monarch
Atrial fibrillation Pathology Pathological finding Moderate HPO, Monarch
Atrial flutter Pathology Pathological finding Moderate HPO, Monarch
Atrial septal defect Pathology Pathological finding Moderate HPO, Monarch
Atrioventricular block Pathology Pathological finding Moderate HPO, Monarch
Autism Pathology Pathological finding Moderate HPO, Monarch
Bronchial artery dilatation Pathology Pathological finding Moderate HPO, Monarch
Bronchiolitis Pathology Pathological finding Moderate HPO, Monarch
Cardiac arrest Pathology Pathological finding Moderate HPO, Monarch
Chest pain Pathology Pathological finding Moderate HPO, Monarch
Chest tightness Pathology Pathological finding Moderate HPO, Monarch
Chronic pulmonary obstruction Pathology Pathological finding Moderate HPO, Monarch
Complete right bundle branch block Pathology Pathological finding Moderate HPO, Monarch
Congestive heart failure Pathology Pathological finding Moderate HPO, Monarch
Cough Pathology Pathological finding Moderate HPO, Monarch
Cyanosis Pathology Pathological finding Moderate HPO, Monarch
Decreased DLCO Pathology Pathological finding Moderate HPO, Monarch
Dyspnea Pathology Pathological finding Moderate HPO, Monarch
Easy fatigability Pathology Pathological finding Moderate HPO, Monarch
Elevated circulating NT-proBNP concentration Lab / Clinical Lab value Moderate HPO, Monarch
Elevated pulmonary artery pressure Lab / Clinical Lab value Moderate HPO, Monarch
Elevated right atrial pressure Lab / Clinical Lab value Moderate HPO, Monarch
Epistaxis Pathology Pathological finding Moderate HPO, Monarch
Exertional dyspnea Pathology Pathological finding Moderate HPO, Monarch
Failure to thrive Pathology Pathological finding Moderate HPO, Monarch
Fatigue Functional Functional test Moderate HPO, Monarch
First degree atrioventricular block Pathology Pathological finding Moderate HPO, Monarch
Ground-glass opacification Pathology Pathological finding Moderate HPO, Monarch
Hemoptysis Pathology Pathological finding Moderate HPO, Monarch
Hemosiderin-laden macrophages in bronchoalveolar fluid Pathology Pathological finding Moderate HPO, Monarch
Hypercoagulability Pathology Pathological finding Moderate HPO, Monarch
Hypertension Pathology Pathological finding Moderate HPO, Monarch
Hypertrophic cardiomyopathy Pathology Pathological finding Moderate HPO, Monarch
Hypothyroidism Pathology Pathological finding Moderate HPO, Monarch
Hypoxemia Pathology Pathological finding Moderate HPO, Monarch
Incomplete right bundle branch block Pathology Pathological finding Moderate HPO, Monarch
Increased pulmonary capillary wedge pressure Pathology Pathological finding Moderate HPO, Monarch
Increased pulmonary vascular resistance Pathology Pathological finding Moderate HPO, Monarch
Interrupted inferior vena cava with azygous continuation Pathology Pathological finding Moderate HPO, Monarch
Juvenile rheumatoid arthritis Pathology Pathological finding Moderate HPO, Monarch
Left Isomerism Pathology Pathological finding Moderate HPO, Monarch
Left ventricular systolic dysfunction Pathology Pathological finding Moderate HPO, Monarch
Leukemia Pathology Pathological finding Moderate HPO, Monarch
Muscle weakness Functional Functional test Moderate HPO, Monarch
Obstructive sleep apnea Pathology Pathological finding Moderate HPO, Monarch
Patent ductus arteriosus Pathology Pathological finding Moderate HPO, Monarch
Patent foramen ovale Pathology Pathological finding Moderate HPO, Monarch
Persistent patent ductus venosus Pathology Pathological finding Moderate HPO, Monarch
Premature birth Pathology Pathological finding Moderate HPO, Monarch
Pulmonary arterial hypertension Pathology Pathological finding Moderate HPO, Monarch
Pulmonary arterial hypertension with lack of acute response to NO challenge Pathology Pathological finding Moderate HPO, Monarch
Pulmonary arterial hypertension with positive acute response to NO challenge Pathology Pathological finding Moderate HPO, Monarch
Pulmonary arterial medial hypertrophy Pathology Pathological finding Moderate HPO, Monarch
Pulmonary artery vasoconstriction Pathology Pathological finding Moderate HPO, Monarch
Pulmonary aterial intimal fibrosis Pathology Pathological finding Moderate HPO, Monarch
Pulmonary capillary hemangiomatosis Pathology Pathological finding Moderate HPO, Monarch
Pulmonic stenosis Pathology Pathological finding Moderate HPO, Monarch
Reduced systolic function Lab / Clinical Lab value Moderate HPO, Monarch
Respiratory insufficiency Pathology Pathological finding Moderate HPO, Monarch
Right atrial enlargement Pathology Pathological finding Moderate HPO, Monarch
Right axis deviation Pathology Pathological finding Moderate HPO, Monarch
Right ventricular dilatation Pathology Pathological finding Moderate HPO, Monarch
Right ventricular failure Pathology Pathological finding Moderate HPO, Monarch
Right ventricular hypertrophy Pathology Pathological finding Moderate HPO, Monarch
Stroke Pathology Pathological finding Moderate HPO, Monarch
Syncope Pathology Pathological finding Moderate HPO, Monarch
Telangiectasia Pathology Pathological finding Moderate HPO, Monarch
Ventricular septal defect Pathology Pathological finding Moderate HPO, Monarch
ABCC8
ATP binding cassette subfamily C member 8
Molecular Gene Emerging Open Targets
ABL1
ABL proto-oncogene 1, non-receptor tyrosine kinase
Molecular Gene Emerging Open Targets
ACE2
angiotensin converting enzyme 2
Molecular Gene Emerging Open Targets
ACVRL1
activin A receptor like type 1
Molecular Gene Emerging Open Targets
ADORA1
adenosine A1 receptor
Molecular Gene Emerging Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug NameMechanismType
ACETAZOLAMIDE Gene-targeted
ACETAZOLAMIDE None Gene-targeted
ACETOHEXAMIDE blocker Gene-targeted
ACYCLOVIR Gene-targeted
ADENOSINE agonist; agonist Gene-targeted
ADENOSINE TRIPHOSPHATE Gene-targeted
ALBUTEROL SULFATE agonist Gene-targeted
ALFUZOSIN inhibitor Gene-targeted
ALFUZOSIN HYDROCHLORIDE agonist Gene-targeted
AMINOPHYLLINE inhibitor Gene-targeted
AMIODARONE HYDROCHLORIDE Gene-targeted
AMITRIPTYLINE HYDROCHLORIDE inhibitor Gene-targeted
AMPHETAMINE None Gene-targeted
ARFORMOTEROL Gene-targeted
ARFORMOTEROL TARTRATE agonist Gene-targeted
ASCIMINIB modulator; inhibitor Gene-targeted
ASCIMINIB HYDROCHLORIDE inhibitor Gene-targeted
ASCORBIC ACID Gene-targeted
ASENAPINE Gene-targeted
ASPIRIN Gene-targeted
ATENOLOL inhibitor Gene-targeted
AXITINIB Gene-targeted
AZACITIDINE Gene-targeted
BENZPHETAMINE Gene-targeted
BETAXOLOL inhibitor Gene-targeted
BETHANIDINE Gene-targeted
BICALUTAMIDE None Gene-targeted
BITOLTEROL MESYLATE agonist Gene-targeted
BLINATUMOMAB Gene-targeted
BOSUTINIB inhibitor; inhibitor Gene-targeted
BOSUTINIB MONOHYDRATE inhibitor Gene-targeted
BUSULFAN Gene-targeted
CABERGOLINE inhibitor Gene-targeted
CABOZANTINIB S-MALATE Gene-targeted
CAFFEINE inhibitor; inhibitor Gene-targeted
CAFFEINE CITRATE inhibitor Gene-targeted
CARBACHOL None Gene-targeted
CARTEOLOL Gene-targeted
CARTEOLOL HYDROCHLORIDE inhibitor Gene-targeted
CARVEDILOL inhibitor; inhibitor 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.

DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
SIROLIMUS Small molecule Approved Strong 100
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 DGIdb
ACETAZOLAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
ACETOHEXAMIDE Repurposing Small molecule Approved Strong 95 DGIdb
ACYCLOVIR Repurposing Small molecule Approved Strong 95 DGIdb
ADENOSINE Repurposing Small molecule Approved Strong 95 DGIdb
ADENOSINE TRIPHOSPHATE Repurposing Small molecule Approved Strong 95 DGIdb
ALBUTEROL SULFATE Repurposing Small molecule Approved Strong 95 DGIdb
ALFUZOSIN Repurposing Small molecule Approved Strong 95 DGIdb
ALFUZOSIN HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 DGIdb
AMINOPHYLLINE Repurposing Small molecule Approved Strong 95 DGIdb
AMIODARONE HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 DGIdb
AMITRIPTYLINE HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 DGIdb
AMPHETAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
ARFORMOTEROL Repurposing Small molecule Approved Strong 95 DGIdb
ARFORMOTEROL TARTRATE Repurposing Small molecule Approved Strong 95 DGIdb
ASCIMINIB Repurposing Small molecule Approved Strong 95 DGIdb
ASCIMINIB HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 DGIdb
ASCORBIC ACID Repurposing Small molecule Approved Strong 95 DGIdb
ASENAPINE Repurposing Small molecule Approved Strong 95 DGIdb
ASPIRIN Repurposing Small molecule Approved Strong 95 DGIdb
ATENOLOL Repurposing Small molecule Approved Strong 95 DGIdb
AXITINIB Repurposing Small molecule Approved Strong 95 DGIdb
AZACITIDINE Repurposing Small molecule Approved Strong 95 DGIdb
BENZPHETAMINE Repurposing Small molecule Approved Strong 95 DGIdb
BETAXOLOL Repurposing Small molecule Approved Strong 95 DGIdb
BETHANIDINE Repurposing Small molecule Approved Strong 95 DGIdb
BICALUTAMIDE Repurposing Small molecule Approved Strong 95 ChEMBL
BITOLTEROL MESYLATE Repurposing Small molecule Approved Strong 95 DGIdb
BLINATUMOMAB Repurposing Small molecule Approved Strong 95 DGIdb
BOSUTINIB Repurposing Small molecule Approved Strong 95 DGIdb
BOSUTINIB MONOHYDRATE Repurposing Small molecule Approved Strong 95 DGIdb
BUSULFAN Repurposing Small molecule Approved Strong 95 DGIdb
CABERGOLINE Repurposing Small molecule Approved Strong 95 DGIdb
CABOZANTINIB S-MALATE Repurposing Small molecule Approved Strong 95 DGIdb
CAFFEINE Repurposing Small molecule Approved Strong 95 DGIdb
CAFFEINE CITRATE Repurposing Small molecule Approved Strong 95 DGIdb
CARBACHOL Repurposing Small molecule Approved Strong 95 ChEMBL
CARTEOLOL Repurposing Small molecule Approved Strong 95 DGIdb
CARTEOLOL HYDROCHLORIDE Repurposing Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
CARVEDILOL Repurposing Small molecule Approved Strong 95
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
No indexed hits
View evidence
DGIdb
CELECOXIB Repurposing Small molecule Approved Strong 95 DGIdb
CELIPROLOL Repurposing Small molecule Approved Strong 95 DGIdb
CHLORPROPAMIDE Repurposing Small molecule Approved Strong 95 DGIdb
CIPROFLOXACIN Repurposing Small molecule Approved Strong 95 ChEMBL
CLOMIPRAMINE Repurposing Small molecule Approved Strong 95 DGIdb
CLOMIPRAMINE Repurposing Small molecule Approved Strong 95 ChEMBL
CLOTRIMAZOLE Repurposing Small molecule Approved Strong 95 DGIdb
CLOZAPINE Repurposing Small molecule Approved Strong 95 DGIdb
DrugTypePhaseEvidence ScoreSourcesLinks
ALBUTEROL Small molecule Phase III Moderate 80 Open Targets
AV-101 Small molecule Phase III Moderate 80 Open Targets
BARDOXOLONE METHYL Small molecule Phase III Moderate 80 Open Targets
BERAPROST Small molecule Phase III Moderate 80 Open Targets
EPOPROSTENOL Small molecule Phase III Moderate 80 Open Targets
ESUBERAPROST Small molecule Phase III Moderate 80 Open Targets
ESUBERAPROST SODIUM Small molecule Phase III Moderate 80 Open Targets
IMATINIB Small molecule Phase III Moderate 80 Open Targets
IMATINIB MESYLATE Small molecule Phase III Moderate 80 Open Targets
IPRATROPIUM BROMIDE Small molecule Phase III Moderate 80 Open Targets
MELATONIN Small molecule Phase III Moderate 80 Open Targets
MILRINONE Small molecule Phase III Moderate 80 Open Targets
RALINEPAG Small molecule Phase III Moderate 80 Open Targets
RANOLAZINE Small molecule Phase III Moderate 80 Open Targets
ROSUVASTATIN Small molecule Phase III Moderate 80 Open Targets
SERALUTINIB Small molecule Phase III Moderate 80 Open Targets
SILDENAFIL Small molecule Phase III Moderate 80 Open Targets
SITAXENTAN SODIUM Small molecule Phase III Moderate 80 Open Targets
TREPROSTINIL DIOLAMINE Small molecule Phase III Moderate 80 Open Targets
TREPROSTINIL PALMITIL Small molecule Phase III Moderate 80 Open Targets
TREPROSTINIL SODIUM Small molecule Phase III Moderate 80 Open Targets
VARDENAFIL Small molecule Phase III Moderate 80 Open Targets
2-METHOXYESTRADIOL Small molecule Phase II Emerging 60 Open Targets
ANASTROZOLE Small molecule Phase II Emerging 60 Open Targets
APABETALONE Small molecule Phase II Emerging 60 Open Targets
ASPIRIN Small molecule Phase II Emerging 60 Open Targets
ATORVASTATIN Small molecule Phase II Emerging 60 Open Targets
BENZBROMARONE Small molecule Phase II Emerging 60 Open Targets
BQ-123 Biologic Phase II Emerging 60 Open Targets
CARVEDILOL Small molecule Phase II Emerging 60 Open Targets
CICLETANINE Small molecule Phase II Emerging 60 Open Targets
CXA-10 Small molecule Phase II Emerging 60 Open Targets
DAPAGLIFLOZIN Small molecule Phase II Emerging 60 Open Targets
EMPAGLIFLOZIN Small molecule Phase II Emerging 60 Open Targets
ENRASENTAN Small molecule Phase II Emerging 60 Open Targets
FAMOTIDINE Small molecule Phase II Emerging 60 Open Targets
FLUOXETINE Small molecule Phase II Emerging 60 Open Targets
FRESPACIGUAT Small molecule Phase II Emerging 60 Open Targets
FULVESTRANT Small molecule Phase II Emerging 60 Open Targets
GLUTAMINE Small molecule Phase II Emerging 60 Open Targets
DrugTypePhaseEvidence ScoreTrials & literatureSourcesLinks
ACETAZOLAMIDE Repurposing via AQP1 Small molecule Approved Strong 95 DGIdb
ACETAZOLAMIDE Repurposing via ABCC8 Small molecule Approved Strong 95 ChEMBL
ACETOHEXAMIDE Repurposing via ABCC8 Small molecule Approved Strong 95 DGIdb
ACYCLOVIR Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
ADENOSINE Repurposing via ADORA1 Small molecule Approved Strong 95 DGIdb
ADENOSINE TRIPHOSPHATE Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
ALBUTEROL SULFATE Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
ALFUZOSIN Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
ALFUZOSIN HYDROCHLORIDE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
AMINOPHYLLINE Repurposing via ADORA1 Small molecule Approved Strong 95 DGIdb
AMIODARONE HYDROCHLORIDE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
AMITRIPTYLINE HYDROCHLORIDE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
AMPHETAMINE Repurposing via ABCC8 Small molecule Approved Strong 95 ChEMBL
ARFORMOTEROL Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
ARFORMOTEROL TARTRATE Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
ASCIMINIB Repurposing via BCR Small molecule Approved Strong 95 DGIdb
ASCIMINIB HYDROCHLORIDE Repurposing via BCR Small molecule Approved Strong 95 DGIdb
ASCORBIC ACID Repurposing via AQP1 Small molecule Approved Strong 95 DGIdb
ASENAPINE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
ASPIRIN Repurposing via ADORA1 Small molecule Approved Strong 95 DGIdb
ATENOLOL Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
AXITINIB Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
AZACITIDINE Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
BENZPHETAMINE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
BETAXOLOL Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
BETHANIDINE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
BICALUTAMIDE Repurposing via ABCC8 Small molecule Approved Strong 95 ChEMBL
BITOLTEROL MESYLATE Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
BLINATUMOMAB Repurposing via BCR Small molecule Approved Strong 95 DGIdb
BOSUTINIB Repurposing via BCR Small molecule Approved Strong 95 DGIdb
BOSUTINIB MONOHYDRATE Repurposing via BCR Small molecule Approved Strong 95 DGIdb
BUSULFAN Repurposing via BCR Small molecule Approved Strong 95 DGIdb
CABERGOLINE Repurposing via ADRA1A Small molecule Approved Strong 95 DGIdb
CABOZANTINIB S-MALATE Repurposing via ABL1 Small molecule Approved Strong 95 DGIdb
CAFFEINE Repurposing via ADORA1 Small molecule Approved Strong 95 DGIdb
CAFFEINE CITRATE Repurposing via ADORA1 Small molecule Approved Strong 95 DGIdb
CARBACHOL Repurposing via ABCC8 Small molecule Approved Strong 95 ChEMBL
CARTEOLOL Repurposing via ADRB2 Small molecule Approved Strong 95 DGIdb
CARTEOLOL HYDROCHLORIDE Repurposing via ADRB3 Small molecule Approved Strong 95
No indexed hits
View evidence
DGIdb
CARVEDILOL Repurposing via ADRB3 Small molecule Approved Strong 95
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 ScoreTrials & literatureSourcesLinks
Vitamin C Natural product Nutraceutical Phase I Emerging 10
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
3 pubs
View evidence
Omega-3 Fatty Acids Natural product Nutraceutical Phase II Emerging 10
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
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
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
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
19 pubs
View evidence

Published literature

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

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.