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Disease

Pulmonary arterial hypertension

Late-stage therapeutic development
Also known as PAH, PPH, idiopathic pulmonary hypertension, PAH with overt features of venous/capillaries involvement+1 more

PAH, PPH, idiopathic pulmonary hypertension, PAH with overt features of venous/capillaries involvement, PVOD/PCH.

22
Clinical trials
12
Associated genes
12
Related proteins
Current focus
Endothelin-1 biologyProstacyclin biologyTherapeutic developmentDisease mechanisms & pathology
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 1 regulatory approval from EMA on record.
  • 4 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
Major developments
Upcoming trial readoutHigh impact
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety and Efficacy of L606 (Treprostinil Liposome Inhalation Suspension) in Participants With Pulmonary Hypertension Associated With Interstitial Lung Disease (WHO Group 3)
Results expected Q3 20292026-06-29
Upcoming trial readoutHigh impact
Effect of the Traditional Chinese Medicine Yufeng Ningxin in Patients With Hypertension: a Randomized, Double-blind, Placebo-controlled Trial
Results expected Q4 20272026-06-02
CHMP positive opinion (EU decision pending)Important
CHMP positive opinion — Riociguat
Treatment of Chronic thromboembolic pulmonary hypertension (CTEPH) in adults and treatment of Pulmonary arterial hypertension (PAH) in adults and children from 6 years of age.2026-07-20
Clinical Milestones8View
Results expected 4
Other updates 4
Regulatory Updates2View
  • 2026-07-20CHMP positive opinion — RiociguatTreatment of Chronic thromboembolic pulmonary hypertension (CTEPH) in adults and treatment of Pulmonary arterial hypertension (PAH) in adults and children from 6 years of age.
  • 2025-09-24Approval — MacitentanAdultsMacitentan AccordPharma, as monotherapy or in combination, is indicated for the long-term treatmentof pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III (seesection 5.1)Paediatric populationMacitentan AccordPharma, as monotherapy or in combination, is indicated for the long-term treatmentof pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years andbodyweight ≥ 40 kg with WHO Functional Class (FC) II to III (see section 5.1)
Activity timeline10

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Macitentanapproved

Approval — AdultsMacitentan AccordPharma, as monotherapy or in combination, is indicated for the lon… (2025)

Sotaterceptapproved

Approval — Winrevair, in combination with other pulmonary arterial hypertension (PAH) therapies, is… (2024)

Ambrisentanapproved

Approval — Ambrisentan Mylan is indicated for the treatment of pulmonary arterial hypertension (PAH)… (2019)

Tadalafilapproved

Approval — Talmanco is indicated in adults for the treatment of pulmonary arterial hypertension (PAH… (2017)

Selexipagapproved

Approval — Uptravi is indicated for the long-term treatment of pulmonary arterial hypertension (PAH)… (2016)

Sildenafilapproved

Approval — AdultsTreatment of adult patients with pulmonary arterial hypertension classified as WHO… (2016)

Sildenafil citrateapproved

Approval — Adults Treatment of adult patients with pulmonary arterial hypertension classified as WH… (2016)

Riociguatapproved

Approval — Chronic thromboembolic pulmonary hypertension (CTEPH) Adempas is indicated for the treatm… (2014)

Clinical trials

13 sponsors · 4 new · 0 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme
22
All trials
5
Active
16
Late-stage
5
Completed
Late-stage studies

A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety and Efficacy of L606 (Treprostinil Liposome Inhalation Suspension) in Participants With Pulmonary Hypertension Associated With Interstitial Lung Disease (WHO Group 3)

Phase 3 · Recruiting · Liquidia Technologies, Inc.

A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety and Tolerability of Liposomal Treprostinil Inhalation Suspension (L606) in Subjects With Pulmonary Arterial Hypertension or Pulmonary Hypertension Associated With Interstitial Lung Disease

Phase 3 · Active, not recruiting · Liquidia Technologies, Inc.

A Randomized, Double-blind, Placebo-controlled, Multicentre Trial, Assessing the Impact of Ferric Carboxymaltose on Exercise Capacity and Functional Status in Pulmonary Hypertension

Phase 4 · Recruiting · Ziekenhuis Oost-Limburg

Effect of the Traditional Chinese Medicine Yufeng Ningxin in Patients With Hypertension: a Randomized, Double-blind, Placebo-controlled Trial

Phase 4 · Recruiting · Beijing Anzhen Hospital

An Adaptive, 2-Part, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of IKT-001 in Pulmonary Arterial Hypertension (PAH)

Phase 3 · Recruiting · Inhibikase Therapeutics

A Randomized, Double-Blind, Phase IV Clinical Study to Evaluate the Antihypertensive Efficacy and Changes of Neurohormonal Markers of Fimasartan and Atenolol With Exaggerated Blood Pressure Response During Exercise in Essential Hypertensive Patients

Phase 4 · Completed · Boryung Pharmaceutical Co., Ltd
Recruiting

A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety and Efficacy of L606 (Treprostinil Liposome Inhalation Suspension) in Participants With Pulmonary Hypertension Associated With Interstitial Lung Disease (WHO Group 3)

Phase 3 · Recruiting · Liquidia Technologies, Inc.

A Randomized, Double-blind, Placebo-controlled, Multicentre Trial, Assessing the Impact of Ferric Carboxymaltose on Exercise Capacity and Functional Status in Pulmonary Hypertension

Phase 4 · Recruiting · Ziekenhuis Oost-Limburg

Effect of the Traditional Chinese Medicine Yufeng Ningxin in Patients With Hypertension: a Randomized, Double-blind, Placebo-controlled Trial

Phase 4 · Recruiting · Beijing Anzhen Hospital

An Adaptive, 2-Part, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of IKT-001 in Pulmonary Arterial Hypertension (PAH)

Phase 3 · Recruiting · Inhibikase Therapeutics
Recently completed

A Randomized, Double-Blind, Phase IV Clinical Study to Evaluate the Antihypertensive Efficacy and Changes of Neurohormonal Markers of Fimasartan and Atenolol With Exaggerated Blood Pressure Response During Exercise in Essential Hypertensive Patients

Phase 4 · Completed · Boryung Pharmaceutical Co., Ltd

A Randomized, Multicenter, Parallel Design Phase III Clinical Study to Evaluate the Efficacy and Safety of the Combination of Lercanidipine/Valsartan in Hypertensive Patients Who Are Not Adequately Controlled on Lercanidipine 10mg Monotherapy.

Phase 3 · Completed · LG Life Sciences

An Eight-week Randomised Double-blind Study to Compare the Efficacy and Safety of Telmisartan 80mg Plus Amlodipine 5 mg Fixed-dose Combination vs. Telmisartan 40 mg Plus Amlodipine 5 mg Fixed-dose Combination in Patients With Hypertension

Phase 3 · Completed · Boehringer Ingelheim

An Open-label Study to Evaluate the Antihypertensive Effects of the Fixed-dose Combination of Telmisartan 80 mg and Amlodipine 5 mg (T80/A5) Given Once Daily by 24 h ABPM in Patients With Moderate to Severe Hypertension

Phase 3 · Completed · Boehringer Ingelheim

An Open-label, Multicenter Study to Evaluate the Efficacy and Safety of a 4 Week Therapy With the Single Pill (SPC) Combination of Aliskiren 300 mg / Amlodipine 10 mg in Hypertensive Patients Not Adequately Respond to an Uptitrated 4 Week Therapy With the SPC of Olmesartan 40 mg / Amlodipine 10 mg, With a Potential Extension if Patients Still Not Adequately Respond With a 4 Week Therapy With the SPC Aliskiren 300 mg / Amlodipine 10 mg / HCTZ 12,5 mg

Phase 3 · Completed · Novartis

Evidence coverage

8 treatments

How much of this condition's readable clinical evidence the confidence engine has incorporated, across its most-studied treatments. This measures coverage of the evidence base — not whether any treatment works.

Substantial evidence coverage
66% · 73/111 eligible items incorporated
lowers systolic blood pressure in hypertension65%
lowers diastolic blood pressure in hypertension65%

Largest gap: no meaningful change (9, trial-readability).

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2025emaApproval — Macitentan· AdultsMacitentan AccordPharma, as monotherapy or in combination, is indicated for the long-term treatmentof pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III (seesection 5.1)Paediatric populationMacitentan AccordPharma, as monotherapy or in combination, is indicated for the long-term treatmentof pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years andbodyweight ≥ 40 kg with WHO Functional Class (FC) II to III (see section 5.1) source ↗
2024emaApproval — Sotatercept· Winrevair, in combination with other pulmonary arterial hypertension (PAH) therapies, is indicated for the treatment of PAH in adult patients with WHO Functional Class (FC) II, III and IV. source ↗
2019emaApproval — Ambrisentan· Ambrisentan Mylan is indicated for the treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III, including use in combination treatment. Efficacy has been shown in idiopathic PAH (IPAH) and in PAH associated with connective tissue disease. Ambrisentan Mylan is indicated for the treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III, including use in combination treatment. Efficacy has been shown in idiopathic PAH (IPAH) and in PAH associated with connective tissue disease. source ↗
2017emaApproval — Tadalafil· Talmanco is indicated in adults for the treatment of pulmonary arterial hypertension (PAH) classified as WHO functional class II and III, to improve exercise capacity. Efficacy has been shown in idiopathic PAH (IPAH) and in PAH related to collagen vascular disease. source ↗
2016emaApproval — Sildenafil citrate· Adults Treatment of adult patients with pulmonary arterial hypertension classified as WHO functional class II and III, to improve exercise capacity. Efficacy has been shown in primary pulmonary hypertension and pulmonary hypertension associated with connective tissue disease. Paediatric population Treatment of paediatric patients aged 1 year to 17 years old with pulmonary arterial hypertension. Efficacy in terms of improvement of exercise capacity or pulmonary haemodynamics has been shown in primary pulmonary hypertension and pulmonary hypertension associated with congenital heart disease. source ↗
2016emaApproval — Sildenafil· AdultsTreatment of adult patients with pulmonary arterial hypertension classified as WHO functional class II and III, to improve exercise capacity. Efficacy has been shown in primary pulmonary hypertension and pulmonary hypertension associated with connective tissue disease. Paediatric populationTreatment of paediatric patients aged 1 year to 17 years old with pulmonary arterial hypertension. Efficacy in terms of improvement of exercise capacity or pulmonary haemodynamics has been shown in primary pulmonary hypertension and pulmonary hypertension associated with congenital heart disease (see section 5.1). source ↗
2016emaApproval — Selexipag· Uptravi is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients with WHO functional class (FC) II–III, either as combination therapy in patients insufficiently controlled with an endothelin receptor antagonist (ERA) and/or a phosphodiesterase type 5 (PDE-5) inhibitor, or as monotherapy in patients who are not candidates for these therapies. Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease. source ↗
2014emaApproval — Riociguat· Chronic thromboembolic pulmonary hypertension (CTEPH) Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with inoperable CTEPH, persistent or recurrent CTEPH after surgical treatment, to improve exercise capacity. Pulmonary arterial hypertension (PAH) AdultsAdempas, as monotherapy or in combination with endothelin receptor antagonists, is indicated for the treatment of adult patients with pulmonary arterial hypertension (PAH) with WHO Functional Class (FC) II to III to improve exercise capacity. Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease. PaediatricsAdempas is indicated for the treatment of PAH in paediatric patients aged less than 18 years of age and body weight ? 50 kg with WHO Functional Class (FC) II to III in combination with endothelin receptor antagonists. source ↗
Other regulatory activity
2026emaCHMP positive opinion — Riociguat· Treatment of Chronic thromboembolic pulmonary hypertension (CTEPH) in adults and treatment of Pulmonary arterial hypertension (PAH) in adults and children from 6 years of age. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

BMPR20.75ATP13A30.63EDNRB0.62EDNRA0.62ACVRL10.61PTGIR0.61PDE5A0.61GUCY1A10.60INHBA0.59GUCY1B10.58GUCY1A20.58SMAD90.56

Disease biology

12 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Endothelin receptor type Bvia EDNRB0.62Endothelin-1 receptorvia EDNRA0.62Prostacyclin receptorvia PTGIR0.61cGMP-specific 3',5'-cyclic phosphodiesterasevia PDE5A0.61Guanylate cyclase soluble subunit alpha-1via GUCY1A10.60Inhibin beta A chainvia INHBA0.59Guanylate cyclase soluble subunit beta-1via GUCY1B10.58Guanylate cyclase soluble subunit alpha-2via GUCY1A20.58Potassium channel subfamily K member 3via KCNK30.50Carbamoyl-phosphate synthase [ammonia], mitochondrialvia CPS10.47Apelin receptorvia APLNR0.10Mitogen-activated protein kinase kinase kinase 5via MAP3K50.10

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A progressive rare pulmonary disease characterized by high blood pressure in the PULMONARY ARTERY.

Synonyms

PAH, PPH, idiopathic pulmonary hypertension, PAH with overt features of venous/capillaries involvement, PVOD/PCH

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.