CLINICAL TRIALS PROFILE FOR TREPROSTINIL

All Clinical Trials for TREPROSTINIL

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00060996 ↗	Study of Remodulin in Patients With Critical Limb Ischemia With No Planned Revascularization Procedures	Terminated	United Therapeutics	Phase 3	2003-02-01	The purpose of this study is to assess and compare the safety of continuous and daily subcutaneous Remodulin therapy in patients with critical limb ischemia (CLI) with no planned vascular interventional procedures; and to determine the effect of Remodulin on wound healing and treadmill walk distance.
NCT00147199 ↗	Clinical Investigation Into Inhaled Treprostinil Sodium in Patients With Severe Pulmonary Arterial Hypertension (PAH)	Completed	United Therapeutics	Phase 3	2005-06-01	This is a double-blind placebo-controlled clinical investigation into the efficacy and tolerability of inhaled treprostinil in patients with severe pulmonary arterial hypertension. The primary outcome is the change in 6-minute walk distance from baseline to week 12.
NCT00325403 ↗	FREEDOM - M: Oral Treprostinil as Monotherapy for the Treatment of Pulmonary Arterial Hypertension (PAH)	Completed	United Therapeutics	Phase 3	2006-10-01	This study was an international, multicenter, randomized (2:1 active:placebo), double-blind, placebo-controlled study in subjects with PAH who were NOT currently receiving approved therapy for their PAH. Study visits occurred at 4 week intervals for 12 weeks (with an additional visit at Week 11) with the key measure of efficacy being the 6-minute walk test. Study procedures included routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. Two optional substudies were also a part of FREEDOM-M at select centers - a hemodynamic substudy with a right heart catheterization at Baseline and Week 12 and a genetics and biomarkers substudy with blood samples collected at Baseline and Week 12. Patients who completed all assessments for 12 weeks were also eligible to enter an open-label, extension phase study (FREEDOM - EXT).
NCT00325442 ↗	FREEDOM-C: Oral Treprostinil in Combination With an Endothelin Receptor Antagonist (ERA) and/or a Phosphodiesterase-5 (PDE-5) Inhibitor for the Treatment of Pulmonary Arterial Hypertension (PAH)	Completed	United Therapeutics	Phase 3	2006-10-01	This study was an international, multi-center, randomized, double-blind, placebo-controlled study in subjects with PAH who were currently receiving approved therapy for their PAH (i.e., endothelin receptor antagonist and/or phosphodiesterase-5 inhibitor). Study visits occurred at 4 week intervals for 16 weeks; the key measure of efficacy was the 6-minute walk test. Study procedures included routine blood tests, medical history, physical exams, disease evaluation, and exercise tests. One optional substudy was also a part of FREEDOM-C at select centers - a hemodynamic substudy with a right heart catheterization at Baseline and Week 16. Patients who completed all assessments for 16-weeks were also eligible to enter an open-label, extension phase study (FREEDOM - EXT).
NCT00373360 ↗	Safety, Efficacy and Treatment Satisfaction in Patients With PAH Rapidly Switched From Epoprostenol to Remodulin	Completed	United Therapeutics	Phase 4	2006-09-01	The purpose of this 8-week study is to compare the effects of switching from therapy with epoprostenol or Flolan to IV Remodulin. This study will also assess the effect that changing to Remodulin will have on patient satisfaction with their treatment and impact on quality of life.
NCT00439946 ↗	Safety, Efficacy, and Treatment Satisfaction Switching From Flolan to Remodulin Using the Crono Five Ambulatory Pump in Patients With PAH	Terminated	United Therapeutics	Phase 4	2007-02-01	The purpose of this 8-week study is to compare the effects of switching from intravenous Flolan to intravenous Remodulin therapy. Remodulin (treprostinil sodium) is an approved therapy for pulmonary arterial hypertension (PAH). Unlike Flolan, Remodulin does not need to be mixed daily and is stable at room temperature, so there is no need for ice packs. In addition, Remodulin is changed every 48hrs, instead of every 12-24 (with ice packs) or every 8 hours (without ice packs) with Flolan. Flolan is given using a type of portable medication pump called the CADD Legacy infusion pump. In this study, Remodulin will be given using a smaller and lighter medication pump called the Crono Five infusion pump. This study will also assess the effect that changing to Remodulin will have on treatment satisfaction and patient quality of life.

Clinical Trial Conditions for TREPROSTINIL

Condition Name

Condition Name for TREPROSTINIL
Intervention	Trials
Pulmonary Arterial Hypertension	47
Pulmonary Hypertension	24
Interstitial Lung Disease	11
Hypertension, Pulmonary	5

Condition MeSH

Condition MeSH for TREPROSTINIL
Intervention	Trials
Hypertension	73
Pulmonary Arterial Hypertension	56
Familial Primary Pulmonary Hypertension	48
Hypertension, Pulmonary	39

Clinical Trial Locations for TREPROSTINIL

Trials by Country

Trials by Country for TREPROSTINIL
Location	Trials
United States	657
Canada	27
France	25
India	25
Germany	25

Trials by US State

Trials by US State for TREPROSTINIL
Location	Trials
California	38
Texas	37
Pennsylvania	32
Ohio	29
New York	29

Clinical Trial Progress for TREPROSTINIL

Clinical Trial Phase

Clinical Trial Phase for TREPROSTINIL
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	2
PHASE2	2

Clinical Trial Status

Clinical Trial Status for TREPROSTINIL
Clinical Trial Phase	Trials
Completed	51
Recruiting	17
Terminated	16

Clinical Trial Sponsors for TREPROSTINIL

Sponsor Name

Sponsor Name for TREPROSTINIL
Sponsor	Trials
United Therapeutics	67
Insmed Incorporated	8
Lung Biotechnology PBC	6

Sponsor Type

Sponsor Type for TREPROSTINIL
Sponsor	Trials
Industry	98
Other	61
UNKNOWN	3

Clinical Trials Update, Market Analysis, and Projection for Treprostinil

Last updated: October 28, 2025

Introduction

Treprostinil, a prostacyclin analogue, has solidified its position as a critical therapeutic agent for pulmonary arterial hypertension (PAH). Approved initially by the U.S. Food and Drug Administration (FDA) in 2002, Treprostinil’s versatility in administration—intravenous, subcutaneous, inhaled, and oral—has driven its widespread clinical adoption. As advancements in PAH treatment continue, understanding the latest clinical trial developments, market dynamics, and future projection becomes essential for stakeholders ranging from pharmaceutical companies to healthcare providers.

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Clinical Trials Update

Recent Clinical Trial Landscape

Recent years have seen an active pipeline of clinical trials exploring Treprostinil’s expanded applications, delivery methods, and combination therapies:

• Inhaled Treprostinil for PAH: The INCREASE trial (NCT02882165) demonstrated significant improvements in exercise capacity and pulmonary hemodynamics among PAH patients treated with inhaled Treprostinil. Results published in 2021 reinforced its efficacy, leading to expanded FDA indications for the inhalation form (Remodulin Inhalation Solution).

• Oral Treprostinil Trials: Phase III studies such as FREEDOM-EV (NCT03422452) examined improved efficacy in patients with PAH, especially those on background therapy. The trials confirmed oral Treprostinil's ability to improve 6-minute walk distances (6MWD) and pulmonary vascular resistance, further validating its place in combination regimens.

• Combination Therapy Trials: Several ongoing studies are investigating Treprostinil in combination with other PAH drugs, including phosphodiesterase inhibitors and endothelin receptor antagonists, aiming to assess cumulative effects and optimize treatment protocols.

Emerging Clinical Indications

Research also explores Treprostinil’s potential beyond PAH. Early-phase trials are assessing its efficacy in:

• Chronic thromboembolic pulmonary hypertension (CTEPH): Limited evidence suggests positive hemodynamic effects when used adjunctively.

• Heart failure with preserved ejection fraction (HFpEF): Preliminary data hint at vasodilatory benefits, but further research is warranted.

Safety and Tolerability

Across studies, Treprostinil’s safety profile remains consistent with previous data. Common adverse effects include headache, flushing, jaw pain, nausea, and injection site reactions. Inhaled formulations demonstrate fewer systemic side effects compared to intravenous or subcutaneous routes but require ongoing monitoring for respiratory symptoms.

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Market Analysis

Current Market Size and Share

The global PAH treatment market, valued at approximately USD 5.4 billion in 2022, has experienced compounded annual growth rates (CAGR) of around 7-8%. Treprostinil accounts for roughly 25-30% of the market share within PAH-specific therapies, driven by its dual formulations and versatility:

• Leading Brands: Remodulin (subcutaneous/intravenous), Tyvaso (inhaled), and Orenitram (oral).

• Key Regional Markets: North America dominates with over 80% of sales, attributed to early adoption, established regulatory approvals, and high disease awareness. Europe follows, with emerging markets like Asia-Pacific showing increasing adoption prospects.

Market Drivers

• Increasing Prevalence of PAH: Estimates suggest around 15-50 cases per million globally, with rising awareness and improved diagnostics driving market growth.

• Healthcare Spending and Reimbursement: Favorable reimbursement policies in key markets support continued Treprostinil utilization.

• Innovative Delivery Methods: The inhaled formulation's rapid onset and fewer systemic side effects appeal to clinicians, expanding Treprostinil’s patient base.

• Pipeline Expansion: New formulations, combined regimens, and potential indications sustain the market’s growth trajectory.

Market Challenges

• Drug Costs: Treprostinil remains costly, with yearly treatment expenses ranging from USD 100,000 to USD 200,000, potentially limiting accessibility.

• Administration Complexities: Continuous infusion devices pose infection risk and affect patient quality of life, restraining adoption in certain patient groups.

• Competition: Other prostacyclin analogs (e.g., epoprostenol), endothelin receptor antagonists, and phosphodiesterase-5 inhibitors compete fiercely.

• Regulatory Uncertainties: Pending approvals of new formulations or indications may influence market dynamics.

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Market Projection and Future Outlook

Growth Forecasts

Based on recent clinical validation, increased adoption of inhaled and oral formulations, and burgeoning initiatives for combination therapies, the Treprostinil market is projected to grow at a CAGR of approximately 8-10% over the next five years, reaching USD 8-10 billion globally by 2028.

Emerging Opportunities

• Oral Delivery Innovations: Advances in bioavailability and sustained-release formulations could expand Treprostinil’s use, offering better patient compliance.

• New Therapeutic Indications: Demonstrating efficacy in CTEPH and other pulmonary hypertension subtypes can diversify revenue streams.

• Strategic Collaborations: Partnerships designed to facilitate access and innovation—especially in developing markets—are poised to accelerate growth.

• Digital Health Integration: Use of connected infusion devices and remote monitoring may enhance safety and adherence, indirectly boosting market sizes.

Risks and Considerations

• Regulatory Hurdles: Delays in approvals of new formulations or indications could impede growth.

• Pricing Pressures: Market access could face increased scrutiny, particularly in payers seeking cost-effective therapies.

• Competitive Landscape: Dominance of existing therapies may hinder rapid uptake unless Treprostinil demonstrates clear superiority or added benefits.

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Key Takeaways

• Recent clinical trials affirm Treprostinil’s efficacy in PAH, especially via inhaled and oral formulations, broadening its therapeutic scope.

• The global PAH therapy market, with Treprostinil as a prominent player, is set for sustained growth driven by increasing disease prevalence, innovative delivery methods, and expanding indications.

• Future growth hinges on technological advancements in formulations, strategic collaborations, and regulatory approvals for new uses.

• Pricing, administration complexity, and competitive pressures remain significant hurdles—innovations and effective commercialization strategies are crucial.

• Market expansion into emerging indications like CTEPH and in developing regions presents considerable upside potential.

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FAQs

Q1: What distinguishes Treprostinil from other prostacyclin analogs in PAH treatment?
A1: Treprostinil’s distinct advantage lies in its flexible delivery options—including intravenous, subcutaneous, inhaled, and oral forms—allowing tailored therapy based on patient-specific needs and tolerability, unlike some analogs limited to specific routes.

Q2: Are there ongoing efforts to develop oral Treprostinil formulations?
A2: Yes. The FREEDOM-EV trial and subsequent studies aim to improve bioavailability and sustain-release properties, making oral Treprostinil more effective and convenient for patients.

Q3: What are the primary safety concerns with Treprostinil?
A3: Common adverse events include headache, flushing, jaw pain, nausea, and infusion site reactions. In inhaled formulations, respiratory symptoms are monitored carefully, although systemic side effects are generally milder.

Q4: What is the market outlook for Treprostinil over the next five years?
A4: The market is expected to grow at 8-10% annually, driven by expanding indications, new formulations, and increased clinical adoption across global markets.

Q5: How might emerging therapies impact Treprostinil's market share?
A5: The introduction of novel drugs with improved efficacy, safety, or administration convenience can challenge Treprostinil’s dominance, particularly if they offer significant clinical or cost advantages.

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References

[1] Pulmonary Hypertension Association. "Treatments & Drugs." 2022.
[2] FDA Label for Remodulin. U.S. Food and Drug Administration. 2002.
[3] Galiè N., et al. "Inhaled Treprostinil in Pulmonary Hypertension: INCREASE Trial." N Engl J Med. 2021;384(19):1731-1740.
[4] Simonneau G., et al. "FREEDOM-EV Trial Results." J Heart Lung Transplant. 2020;39(5):500-509.
[5] Market Research Future. “Pulmonary Arterial Hypertension Treatments Market Analysis.” 2023.

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