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Disease

Idiopathic pulmonary arterial hypertension

Late-stage therapeutic development

Also known as IPAH, primary pulmonary arterial hypertension, primary pulmonary hypertension.

22
Clinical trials
12
Associated genes
1
Related proteins
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Approval: Ventavis (EMA)

Regulatory2003-09-15EMA

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
  • 4 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
Clinical Milestones8View
Results expected 4
Other updates 4
Activity timeline8

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
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
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 ↗
2008emaApproval — Tadalafil· AdultsTreatment of pulmonary arterial hypertension (PAH) classified as WHO functional class II and III, to improve exercise capacity (see section 5.1). Efficacy has been shown in idiopathic PAH (IPAH) and in PAH related to collagen vascular disease. Paediatric populationTreatment of paediatric patients aged 2 years and above with pulmonary arterial hypertension (PAH) classified as WHO functional class II and III. source ↗
2008emaApproval — Ambrisentan· Volibris is indicated for treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III, including use in combination treatment (see section 5.1).  Efficacy has been shown in idiopathic PAH (IPAH) and in PAH associated with connective tissue disease. Volibris is indicated for treatment of PAH in adolescents and children (aged 8 to less than 18 years) of WHO Functional Class (FC) II to III including use in combination treatment. Efficacy has been shown in IPAH, familial, corrected congenital and in PAH associated with connective tissue disease (see section 5.1). 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.

BMPR20.68PTGIR0.51EIF2AK40.48SMAD90.47SLC15A40.29SLC4A40.29AFG1L0.24UBE2Q20.12FBXL130.10PM20D10.06BSDC10.00ZNF2210.00

Disease biology

1 match

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

Prostacyclin receptorvia PTGIR0.51

Reference

Authoritative identity, definition & identifiers.

Synonyms

IPAH, primary pulmonary arterial hypertension, primary pulmonary hypertension

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.