OPSUMIT (macitentan)
OPSUMIT is indicated for the treatment of Pulmonary Arterial Hypertension.
How OPSUMIT Works
Macitentan is an endothelin receptor antagonist (ERA) that inhibits the binding of endothelin-1 (ET-1) to both $ET_A$ and $ET_B$ receptors. In PAH, the ET system is upregulated, leading to deleterious effects such as vasoconstriction, fibrosis, and vascular hypertrophy. Macitentan provides dual receptor antagonism with high affinity and sustained occupancy in pulmonary arterial smooth muscle cells. The drug also produces a pharmacologically active metabolite that contributes to the inhibition of ET receptors.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2013-10-18
- Patent Cliff
- 2029
- Revenue
- $580M (Q4-2025)
- Routes
- ORAL
- Dosage Forms
- TABLET
OPSUMIT Approval History
What OPSUMIT Treats
1 indicationsOPSUMIT is approved for 1 conditions since its original approval in 2013. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Pulmonary Arterial Hypertension
OPSUMIT Boxed Warning
EMBRYO-FETAL TOXICITY OPSUMIT is contraindicated for use during pregnancy because it may cause fetal harm based on animal data [see Contraindications (4.1) , Warnings and Precautions (5.1) , Use in Specific Populations (8.1) ]. Therefore, for females of reproductive potential, exclude pregnancy before the start of treatment with OPSUMIT. Advise use of effective contraception before the initiation of treatment, during treatment, and for one month after stopping treatment with OPSUMIT [see Dosage ...
WARNING: EMBRYO-FETAL TOXICITY OPSUMIT is contraindicated for use during pregnancy because it may cause fetal harm based on animal data [see Contraindications (4.1) , Warnings and Precautions (5.1) , Use in Specific Populations (8.1) ]. Therefore, for females of reproductive potential, exclude pregnancy before the start of treatment with OPSUMIT. Advise use of effective contraception before the initiation of treatment, during treatment, and for one month after stopping treatment with OPSUMIT [see Dosage and Administration (2.2) , Use in Specific Populations (8.3) ]. When pregnancy is detected, discontinue OPSUMIT as soon as possible [see Warnings and Precautions (5.1) ]. WARNING: EMBRYO-FETAL TOXICITY See full prescribing information for complete boxed warning. Based on animal data, OPSUMIT may cause fetal harm if used during pregnancy ( 4.1 , 5.1 , 8.1 ). Females of reproductive potential: exclude pregnancy before start of treatment. Prevent pregnancy prior to initiation of treatment, during treatment and for one month after treatment by using effective methods of contraception ( 2.2 , 8.3 ). When pregnancy is detected, discontinue OPSUMIT as soon as possible ( 5.1 ).
OPSUMIT Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in OPSUMIT's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications OPSUMIT treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to OPSUMIT
3 of 18FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
29 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05179876 PLATYPUS | CR109121 NOPRODPAPUH3001, 2021-002297-11 | Ph 3 | recruiting | A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option |
| NCT05167825 results posted | CR109128 67896062PAH3001, 2023-000984-30 | Ph 3 | completed | A Study of Macitentan in Japanese Pediatric Participants With Pulmonary Arterial Hypertension |
| NCT02932410 TOMORROW results posted | AC-055-312 | Ph 3 | completed | A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH) |
| NCT04271475 MACiTEPH results posted | CR108742 67896062CTP3001, 2019-004131-24 | Ph 3 | terminated | A Study to Evaluate Efficacy and Safety of Macitentan 75 mg in Inoperable or Persistent/Recurrent Chronic Thromboembolic Pulmonary Hypertension |
| NCT03359291 | AC-055-122 | Ph 1 | completed | Clinical Study to Investigate the Effect of Macitentan on the Concentrations of Rosuvastatin in the Blood of Healthy Male Subjects |
| NCT01346930 MUSIC OL | AC-055B202 | Ph 2 | withdrawn | Safety and Tolerability Study of Macitentan in Patients With Idiopathic Pulmonary Fibrosis |
| NCT03422328 UMBRELLA | AC-055-314 2017-003934-10 | Ph 3 | completed | A Clinical Study to Investigate the Long-term Safety of the Drug Macitentan in Patients With Pulmonary Hypertension Who Were Previously Treated With Macitentan in Clinical Studies. |
| NCT05731492 | CR109286 2022-002754-74, 67896062PAH1013 | Ph 1 | withdrawn | A Study of Macitentan in Children Below 2 Years of Age |
| NCT04235270 | CR108735 67896062PAH1006 | Ph 1 | completed | A Study of Macitentan and Tadalafil as a Fixed Dose Combination and the Free Combination in Healthy Adult Participants |
| NCT05392530 | CR109202 2022-000275-39, 67896062PAH1012 | Ph 1 | completed | A Study of Two Different Test Formulations Compared to the Reference Formulation of Macitentan in Healthy Adult Participants |
| NCT05433675 | CR109185 67896062PAH1010, 2022-000262-17 | Ph 1 | completed | A Study of Two Macitentan Formulations in Healthy Adult Participants |
| NCT02310672 REPAIR results posted | AC-055-403 | Ph 4 | completed | REPAIR: Right vEntricular Remodeling in Pulmonary ArterIal hypeRtension |
| NCT02558231 TRITON results posted | AC-065A308 | Ph 3 | completed | The Efficacy and Safety of Initial Triple Versus Initial Dual Oral Combination Therapy in Patients With Newly Diagnosed Pulmonary Arterial Hypertension |
| NCT00667823 SERAPHIN OL results posted | AC-055-303 | Ph 3 | completed | Clinical Study to Assess the Long-term Safety and Tolerability of ACT 064992 in Patients With Symptomatic Pulmonary Arterial Hypertension |
| NCT02112487 | AC-055-311 | Ph 3 | completed | Extension of the Psychometric Validation Study ORCHESTRA in Patients With PAH |
| NCT02060721 MERIT-2 results posted | AC-055E202 2013-003457-25 | Ph 2 | completed | Clinical Study to Assess the Safety, Tolerability and Efficacy of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension |
| NCT02382016 PORTICO results posted | AC-055-404 | Ph 4 | completed | PORtopulmonary Hypertension Treatment wIth maCitentan - a randOmized Clinical Trial |
| NCT04211272 | CR108727 2019-004001-27, 67896062PAH1003 | Ph 1 | completed | A 2-part Study to Investigate the Effect of Macitentan in Healthy Male Participants |
| NCT04963439 | CR109027 2021-001258-67, 67896062PAH1008 | Ph 1 | completed | A Study of Two Macitentan Pediatric Formulations in Healthy Adult Participants |
| NCT02021292 MERIT-1 results posted | AC-055E201 | Ph 2 | completed | Clinical Study to Assess the Efficacy, Safety and Tolerability of Macitentan in Subjects With Inoperable Chronic Thromboembolic Pulmonary Hypertension |
| NCT03153111 SERENADE results posted | AC-055G202 2016-003653-15 | Ph 2 | completed | A Study to Evaluate Whether Macitentan is an Effective and Safe Treatment for Patients With Heart Failure With Preserved Ejection Fraction and Pulmonary Vascular Disease |
| NCT05373108 results posted | 18-001544 | Ph 4 | completed | Endothelin-1 and Cardiac Allograft Vasculopathy (CAV) |
| NCT02893176 | 14-001710 | Ph 4 | withdrawn | Macitentan in the Treatment of Organ Rejection After Lung Transplantation |
| NCT02651272 MENSCH results posted | H-33165 | Ph 2 | terminated | Macitentan in Pulmonary Hypertension of Sickle Cell Disease |
| NCT02968901 OPTIMA | AC-055-405 | Ph 4 | terminated | Clinical Study Evaluating the Effects of First-line Oral cOmbination theraPy of maciTentan and tadalafIl in Patients With Newly Diagnosed pulMonary Arterial Hypertension (OPTIMA) |
| NCT02070991 MELODY-1 results posted | AC-055G201 | Ph 2 | completed | Clinical Study to Evaluate the Safety and Tolerability of Macitentan in Subjects With Combined Pre- and Post-capillary Pulmonary Hypertension (CpcPH) Due to Left Ventricular Dysfunction |
| NCT01841762 results posted | AC-055-401 | Ph 3 | completed | Clinical Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument |
| NCT01847014 SYMPHONYext results posted | AC-055-402 | Ph 3 | terminated | Clinical Study of Macitentan in Patients With PAH to Psychometrically Validate PAH-SYMPACT Instrument |
| NCT02081690 ORCHESTRA results posted | AC-055-310 | Ph 3 | terminated | A Pulmonary Arterial Hypertension Study With Macitentan to Validate the PAH-SYMPACT™ in France, Italy and Spain |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
OPSUMIT FDA Label Details
Indications & Usage
FDA Label (PDF)OPSUMIT is indicated for the treatment of Pulmonary Arterial Hypertension.
WARNING: EMBRYO-FETAL TOXICITY OPSUMIT is contraindicated for use during pregnancy because it may cause fetal harm based on animal data [see Contraindications (4.1) , Warnings and Precautions (5.1) , Use in Specific Populations (8.1) ]. Therefore, for females of reproductive potential, exclude pregn...
OPSUMIT Patents & Exclusivity
Patents (10 active)
Exclusivity
Pro Intelligence Preview
Deep insights for OPSUMIT
Revenue Insights
- • Q4-2025: $580M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2029
- • 10 active patents
Trial Analysis
- • 29 total trials
- • Stage: Declining
Competitive Landscape
- • 18 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment