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macitentan/tadalafil

Phase 3

Pulmonary Arterial Hypertension (PAH) (WHO Group 1 PH) | Small molecule | Cardiovascular |Johnson & Johnson|Last Updated: Dec 19, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment187
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03904693Clinical Study to Compare the Efficacy and Safety of Macitentan and Tadalafil Monotherapies With the Corresponding Fixed-dose Combination Therapy in Subjects With Pulmonary Arterial Hypertension (PAH)PHASE3 COMPLETED 187Jul 29, 2019Sep 27, 2024Dec 19, 2025148 United States, Australia +17
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Study Endpoints
Primary Endpoints
Change in Pulmonary Vascular Resistance (PVR) Expressed as the Ratio of Geometric Means of End of Double-blind Treatment (EDBT) to Baseline
Baseline, EDBT (up to 16 weeks)

Change in PVR expressed as the ratio of geometric means of EDBT to baseline were reported.

Secondary Endpoints
Change From Baseline to EDBT in 6-minutes Walking Distance (6MWD)
Baseline, EDBT (Week 16)
Change From Baseline in Pulmonary Arterial Hypertension Symptoms and Impact (PAH-SYMPACT) in Cardiopulmonary Symptom Domain Scores to EDBT
Baseline, EDBT (Week 16)
Change From Baseline in Pulmonary Arterial Hypertension Symptoms and Impact (PAH-SYMPACT) in Cardiovascular Symptom Domain Scores to EDBT
Baseline, EDBT (Week 16)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
FDC therapy + Placebo macitentan + Placebo tadalafilEXPERIMENTALSubjects to receive FDC macitentan/tadalafil (macitentan 10 mg and tadalafil 40 mg) plus matching placebos for the two other study treatments.
Macitentan mono-therapy + Placebo tadalafil + Placebo FDCACTIVE_COMPARATORSubjects to receive macitentan 10 mg plus matching placebos for the two other study treatments.
Tadalafil mono-therapy + Placebo macitentan + Placebo FDCACTIVE_COMPARATORSubjects to receive tadalafil 40 mg (2 x 20 mg) plus matching placebos for the two other study treatments.
Interventions
NameTypeDescription
FDC macitentan/tadalafilDRUGFilm-coated tablet with 10 mg macitentan and 40 mg tadalafil, to be administered orally once daily.
Macitentan 10 mgDRUGFilm-coated tablet with 10 mg macitentan, to be administered orally once daily.
Tadalafil 40 mgDRUGFilm-coated tablet with 40 mg tadalafil (2 x 20 mg tablets), to be administered orally once daily.
Placebo FDCDRUGMatching placebo not containing any active substance but otherwise identical in appearance to the respective active drug tablet, to be administered orally once daily.
Placebo macitentanDRUGMatching placebo not containing any active substance but otherwise identical in appearance to the respective active drug tablet, to be administered orally once daily.
Placebo tadalafilDRUGMatching placebo not containing any active substance but otherwise identical in appearance to the respective active drug tablet, to be administered orally once daily.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites148

Inclusion Criteria: * Signed and dated informed consent form (ICF) * Confirmed diagnosis of symptomatic PAH in WHO FC II or III * Symptomatic PAH belonging to one of the following subgroups of WHO Group 1 pulmonary hypertension: * Idiopathic * Heritable * Drug- or toxin-induced * Associate...

Countries:United StatesAustraliaBrazilBulgariaCanadaChinaCzechiaGermanyHungaryItalyJapanMalaysiaMexicoPolandRussiaSouth AfricaSpainTaiwanTurkey (Türkiye)
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