Respiratory

Pulmonary Arterial Hypertension

Also known as PAH, primary pulmonary hypertension, BMPR2 mutation, Group 1 pulmonary hypertension

ORPHA:182090 ↗Gene BMPR2 (heritable PAH)Gene ACVRL1 Gene ENG Gene and othersPrevalence 1-9 per 100,000 (Orphanet)Onset All agesGenetic or idiopathic

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Patient organisations

Pulmonary Hypertension AssociationPatient association
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Registry: REVEAL Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

NORD ↗GARD ↗Orphanet ORPHA:182090 ↗

About Pulmonary Arterial Hypertension

Pulmonary arterial hypertension is characterized by progressive obliteration of small pulmonary arteries, leading to increased right heart afterload, right heart failure, and death. About 25% of heritable PAH cases carry BMPR2 mutations. Approved therapies target three pathways (endothelin, prostacyclin, nitric oxide), with triple combination therapy now standard of care. Despite advances, 5-year survival remains approximately 57%.

Common clinical features

Pulmonary arterial hypertensionDyspneaExercise intoleranceFatigueChest painSyncopePeripheral edemaRight heart failure

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

15 approved treatments and 79 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: SildenafilApproved: Treprostinil (Remodulin)Approved: Macitentan (Macitentan component of opsynvi)Approved: Sotatercept (Winrevair)Approved: Beraprost SodiumApproved: Ambrisentan (Ambrisentan mylan)Approved: SitaxentanApproved: Sildenafil Citrate (Granpidam)Approved: Selexipag (Uptravi)Approved: Nitric Oxide (Genosyl)Approved: Iloprost (Aurlumyn)Approved: Bosentan (Stayveer)Approved: Riociguat (Adempas)Approved: Tadalafil (Adcirca)Approved: Epoprostenol Sodium (Epoprostenol sodium)

Phase 3Ranolazine (Aspruzyo sprinkle)

Phase 3Esuberaprost

Phase 3Ipratropium Bromide (Atrovent)

Phase 3Treprostinil Sodium (Trepulmix)

Phase 3Udenafil

Phase 3Treprostinil Diolamine (Orenitram)

Phase 3Imatinib Mesylate (Gleevec)

Phase 3Beraprost

+ 71 more in development

Before you apply

Things trial teams commonly ask about for Pulmonary Arterial Hypertension. Not eligibility rules; those are set by each study.

PAH must be confirmed by right heart catheterization (mPAP above 20 mmHg with PAWP below 15) before any trial
WHO functional class (I through IV) determines trial eligibility - most enroll Class II-III
6-minute walk distance (6MWD) is the most widely used efficacy endpoint in PAH trials

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).