CLINICAL TRIALS PROFILE FOR TYVASO

All Clinical Trials for TYVASO

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
NCT00741819 ↗	Safety Evaluation of Inhaled Treprostinil Administration Following Transition From Inhaled Ventavis in Pulmonary Arterial Hypertension (PAH) Subjects	Completed	United Therapeutics	Phase 4	2008-09-01	This is a 24-month, multi-center, prospective, open-label, safety evaluation in PAH subjects following transition from a stable dose of inhaled iloprost (Ventavis). Subjects are to be evaluated for safety throughout the course of the study while secondary assessments will be conducted at Baseline, Week 6, Week 12, and Months 6, 12, 18 and 24 following initiation of treprostinil sodium.
NCT01266265 ↗	Study of Incidence of Respiratory Tract AEs in Patients Treated With Tyvaso® Compared to Other FDA Approved PAH Therapies	Completed	United Therapeutics		2010-12-01	A surveillance of respiratory tract related adverse events in patients treated with Tyvaso®(treprostinil) Inhalation Solution versus other FDA approved therapies
NCT01268553 ↗	Transition From Injectable Prostacyclin Medication to Inhaled Prostacyclin Medication	Completed	United Therapeutics	Phase 4	2010-08-01	The purpose of this study is to assess tolerability and clinical effects of transition from intravenous (IV, needle in the vein) or subcutaneous (SQ, needle in the skin) to the recently-approved inhaled treprostinil (Tyvaso) for the treatment of pulmonary arterial hypertension (PAH). Our hypothesis is that the transition to inhaled treprostinil will be tolerated by patients. The intravenous and subcutaneous drugs epoprostenol and treprostinil received approval for treatment of PAH many years ago. While these medications improve exercise capacity and the symptoms of PAH, they are given by injection and thus have several side effects, such as pain and catheter infection. This has resulted in many patients either refusing to take the medication or quitting these medications because of not tolerating them. The only other form of prostacyclin treatment available for PAH patients is inhaled. There are 2 inhaled prostacyclins approved for PAH, however one of these requires at least 6 inhalations per day, every day, and takes about 30 minutes to inhale each time. Thus, it has not been a regularly-used medication and issues surrounding compliance make it a riskier drug to use if patients do not get their full doses every day. The other inhaled medication, treprostinil, was approved a few months ago, only needs to be given 4 times a day, and takes about 2-3 minutes to inhale. Since inhaled treprostinil can be administered easily, it is anticipated that many patients will transition from epoprostenol or treprostinil to the recently approved inhaled treprostinil, however we do not know if this is a safe or effective way to manage patients. Thus, the goal of this prospective study is to gather observational data regarding how that switch is made, tolerability of the switch, and, to the extent possible with this methodology, assess clinical effects of the switch. This is a prospective study. Twenty patients > 18 years old with PAH will be enrolled. Patients enrolled will be those in whom a clinical decision to convert from either IV epoprostenol, IV treprostinil, or SQ treprostinil to inhaled treprostinil therapy has been made. This is usually the result of patients asking to switch to inhaled therapy, but only allowed by physicians if they feel the switch would be safe. If eligible, and after informed consent, patients will have a history and physical examination, a 6 min walk test, a cardiopulmonary exercise test (CPET), blood tests, and a symptom questionnaire will be filled out. Patients will then be admitted to the hospital where a monitoring catheter will be placed inside the patient's heart and inhaled treprostinil will be initiated, while the dose of IV/SQ medication is reduced over about 24-26 hours. Clinical follow-up will be at weeks 1, 4, and 12. The procedures above are all part of the routine clinical care that patients would receive if they were to be transitioned to inhaled therapy, including the hospitalization and catheterization. The criteria for them to be able to be switched are conservative. Pressure in their heart and lungs must be low (mPAP < 40 mmHg and RAP
NCT01268553 ↗	Transition From Injectable Prostacyclin Medication to Inhaled Prostacyclin Medication	Completed	Los Angeles Biomedical Research Institute	Phase 4	2010-08-01	The purpose of this study is to assess tolerability and clinical effects of transition from intravenous (IV, needle in the vein) or subcutaneous (SQ, needle in the skin) to the recently-approved inhaled treprostinil (Tyvaso) for the treatment of pulmonary arterial hypertension (PAH). Our hypothesis is that the transition to inhaled treprostinil will be tolerated by patients. The intravenous and subcutaneous drugs epoprostenol and treprostinil received approval for treatment of PAH many years ago. While these medications improve exercise capacity and the symptoms of PAH, they are given by injection and thus have several side effects, such as pain and catheter infection. This has resulted in many patients either refusing to take the medication or quitting these medications because of not tolerating them. The only other form of prostacyclin treatment available for PAH patients is inhaled. There are 2 inhaled prostacyclins approved for PAH, however one of these requires at least 6 inhalations per day, every day, and takes about 30 minutes to inhale each time. Thus, it has not been a regularly-used medication and issues surrounding compliance make it a riskier drug to use if patients do not get their full doses every day. The other inhaled medication, treprostinil, was approved a few months ago, only needs to be given 4 times a day, and takes about 2-3 minutes to inhale. Since inhaled treprostinil can be administered easily, it is anticipated that many patients will transition from epoprostenol or treprostinil to the recently approved inhaled treprostinil, however we do not know if this is a safe or effective way to manage patients. Thus, the goal of this prospective study is to gather observational data regarding how that switch is made, tolerability of the switch, and, to the extent possible with this methodology, assess clinical effects of the switch. This is a prospective study. Twenty patients > 18 years old with PAH will be enrolled. Patients enrolled will be those in whom a clinical decision to convert from either IV epoprostenol, IV treprostinil, or SQ treprostinil to inhaled treprostinil therapy has been made. This is usually the result of patients asking to switch to inhaled therapy, but only allowed by physicians if they feel the switch would be safe. If eligible, and after informed consent, patients will have a history and physical examination, a 6 min walk test, a cardiopulmonary exercise test (CPET), blood tests, and a symptom questionnaire will be filled out. Patients will then be admitted to the hospital where a monitoring catheter will be placed inside the patient's heart and inhaled treprostinil will be initiated, while the dose of IV/SQ medication is reduced over about 24-26 hours. Clinical follow-up will be at weeks 1, 4, and 12. The procedures above are all part of the routine clinical care that patients would receive if they were to be transitioned to inhaled therapy, including the hospitalization and catheterization. The criteria for them to be able to be switched are conservative. Pressure in their heart and lungs must be low (mPAP < 40 mmHg and RAP
NCT01268553 ↗	Transition From Injectable Prostacyclin Medication to Inhaled Prostacyclin Medication	Completed	Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center	Phase 4	2010-08-01	The purpose of this study is to assess tolerability and clinical effects of transition from intravenous (IV, needle in the vein) or subcutaneous (SQ, needle in the skin) to the recently-approved inhaled treprostinil (Tyvaso) for the treatment of pulmonary arterial hypertension (PAH). Our hypothesis is that the transition to inhaled treprostinil will be tolerated by patients. The intravenous and subcutaneous drugs epoprostenol and treprostinil received approval for treatment of PAH many years ago. While these medications improve exercise capacity and the symptoms of PAH, they are given by injection and thus have several side effects, such as pain and catheter infection. This has resulted in many patients either refusing to take the medication or quitting these medications because of not tolerating them. The only other form of prostacyclin treatment available for PAH patients is inhaled. There are 2 inhaled prostacyclins approved for PAH, however one of these requires at least 6 inhalations per day, every day, and takes about 30 minutes to inhale each time. Thus, it has not been a regularly-used medication and issues surrounding compliance make it a riskier drug to use if patients do not get their full doses every day. The other inhaled medication, treprostinil, was approved a few months ago, only needs to be given 4 times a day, and takes about 2-3 minutes to inhale. Since inhaled treprostinil can be administered easily, it is anticipated that many patients will transition from epoprostenol or treprostinil to the recently approved inhaled treprostinil, however we do not know if this is a safe or effective way to manage patients. Thus, the goal of this prospective study is to gather observational data regarding how that switch is made, tolerability of the switch, and, to the extent possible with this methodology, assess clinical effects of the switch. This is a prospective study. Twenty patients > 18 years old with PAH will be enrolled. Patients enrolled will be those in whom a clinical decision to convert from either IV epoprostenol, IV treprostinil, or SQ treprostinil to inhaled treprostinil therapy has been made. This is usually the result of patients asking to switch to inhaled therapy, but only allowed by physicians if they feel the switch would be safe. If eligible, and after informed consent, patients will have a history and physical examination, a 6 min walk test, a cardiopulmonary exercise test (CPET), blood tests, and a symptom questionnaire will be filled out. Patients will then be admitted to the hospital where a monitoring catheter will be placed inside the patient's heart and inhaled treprostinil will be initiated, while the dose of IV/SQ medication is reduced over about 24-26 hours. Clinical follow-up will be at weeks 1, 4, and 12. The procedures above are all part of the routine clinical care that patients would receive if they were to be transitioned to inhaled therapy, including the hospitalization and catheterization. The criteria for them to be able to be switched are conservative. Pressure in their heart and lungs must be low (mPAP < 40 mmHg and RAP
NCT01305252 ↗	A 48-week Study of the Effect of Dual Therapy (Inhaled Treprostinil and Tadafafil) Versus Monotherapy (Tadalafil).	Completed	Northwestern University	Phase 4	2010-07-01	The Study Hypothesis: Aggressive, upfront, dual therapy for treatment-naïve NYHA I/II/III PAH is superior to a traditional "step-up" approach. The study will evaluate: 1. Impact of dual, upfront, therapy on cardiovascular parameters in PAH as gauged by cardiac magnetic resonance imaging (cMRI) at 24 weeks and event free survival at outcome at 48 weeks. 2. Value of novel biomarkers (NT-pro BNP, Mts1/S100A4, and insulin resistance) and cutting-edge imaging technologies (cardiac MRI) as newer endpoints for clinical trials in PAH. 3. Utility of longer clinical trial design with the use of combined clinical events as time to clinical worsening surrogate

Clinical Trial Conditions for TYVASO

Condition Name

Condition Name for TYVASO
Intervention	Trials
Pulmonary Arterial Hypertension	8
Interstitial Lung Disease	7
Pulmonary Hypertension	6
Idiopathic Pulmonary Fibrosis	3

Condition MeSH

Condition MeSH for TYVASO
Intervention	Trials
Hypertension	14
Pulmonary Arterial Hypertension	9
Lung Diseases	9
Familial Primary Pulmonary Hypertension	8

Clinical Trial Locations for TYVASO

Trials by Country

Trials by Country for TYVASO
Location	Trials
United States	214
Canada	3
Israel	2
Puerto Rico	2
Austria	2

Trials by US State

Trials by US State for TYVASO
Location	Trials
North Carolina	13
California	12
Texas	9
Pennsylvania	9
Florida	9

Clinical Trial Progress for TYVASO

Clinical Trial Phase

Clinical Trial Phase for TYVASO
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for TYVASO
Clinical Trial Phase	Trials
Completed	12
Recruiting	4
Not yet recruiting	3

Clinical Trial Sponsors for TYVASO

Sponsor Name

Sponsor Name for TYVASO
Sponsor	Trials
United Therapeutics	17
Lung Biotechnology PBC	2
Bastiaan Driehuys	2

Sponsor Type

Sponsor Type for TYVASO
Sponsor	Trials
Industry	20
Other	15

Clinical Trials Update, Market Analysis, and Projection for Tyvaso (Treprostinil Inhalation Solution)

Last updated: October 28, 2025

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Introduction

Tyvaso (treprostinil inhalation solution) stands as a pivotal medication in the treatment of pulmonary arterial hypertension (PAH), a progressive and life-threatening condition characterized by elevated blood pressure in pulmonary arteries. Recently, the drug has attracted significant attention due to ongoing clinical trials, evolving market dynamics, and prospective growth opportunities. This comprehensive analysis synthesizes recent clinical developments, assesses the current market landscape, and projects future trends, offering vital insights for stakeholders ranging from pharmaceutical companies to investors and healthcare providers.

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Clinical Trials Update

Ongoing and Recent Trials

Tyvaso has been the focus of multiple active clinical trials aimed at expanding its therapeutic scope and optimizing patient outcomes. The most prominent among these is the DURAND study (NCT04849370), a Phase 3 trial exploring the efficacy and safety of Tyvaso in combination therapy for patients with PAH, particularly those who are insufficiently managed with monotherapy. This trial demonstrates a strategic shift toward combination treatments, aligning with broader trends in PAH management.

Additionally, DURAND evaluates the potential of Tyvaso to improve exercise capacity, as measured by the 6-minute walk distance (6MWD), and quality of life metrics, reflecting a focus on patient-centered outcomes. Results are anticipated in late 2023 or early 2024.

FDA and Regulatory Progress

The U.S. Food and Drug Administration (FDA) has approved Tyvaso for the treatment of PAH, emphasizing its role in reducing pulmonary vascular resistance and improving exercise tolerance. Recent interactions suggest ongoing discussions concerning label expansions, particularly for use in other forms of pulmonary hypertension, such as chronic obstructive pulmonary disease (COPD)-associated hypertension, which could extend market potential.

Safety and Efficacy Data

Published data reinforce Tyvaso's safety profile, with adverse events primarily comprising mild respiratory symptoms like cough and throat irritation. Efficacy data from clinical trials consistently demonstrate significant improvements in 6MWD (approximately 20-40 meters) and right ventricular function markers, cementing its standing as a core PAH therapy.

Market Analysis

Current Market Dynamics

The global pulmonary arterial hypertension market was valued at approximately USD 4.2 billion in 2022, with a compound annual growth rate (CAGR) projected at 7.2% from 2023 to 2030. Tyvaso, marketed by United Therapeutics Corporation, captures a notable share within the inhaled prostacyclin segment. Its unique inhalation administration distinguishes it from oral and parenteral counterparts, appealing to patients intolerant of other routes.

Competitive Landscape

The market features key players like Actelion (now part of Johnson & Johnson), Bayer, and Pfizer, offering therapies such as Remodulin (treprostinil IV), Ventavis (iloprost inhalation), and Letairis (ambrisentan). While Tyvaso maintains a competitive edge through its inhalation delivery, emerging therapies with novel mechanisms or sustained-release formulations threaten its growth trajectory.

Market Penetration and Reimbursement

Tyvaso’s penetration is bolstered by favorable reimbursement policies, physician familiarity, and integrated support programs. However, high treatment costs (~USD 37,000 per year) pose barriers, particularly in emerging markets. Efforts to expand access and demonstrate cost-effectiveness are critical to broadening its footprint.

Market Projection

Growth Drivers

• Regulatory Milestones: Positive trial outcomes supporting label expansion could significantly increase prescriptions.
• Patient Preference: Inhalation delivery offers a balance of efficacy and tolerability compared to injectable therapies.
• Combination Therapy Trends: Growing adoption of multi-drug regimens will likely favor Tyvaso as part of combination protocols.
• Global Expansion: Increased focus on Asia-Pacific and Latin America markets offers substantial growth opportunities.

Projected Revenue Outlook

By 2030, the Tyvaso market is expected to reach approximately USD 1.2 billion globally, driven by increased adoption, label extensions, and competitive positioning. The inhaled prostacyclin segment could dominate the PAH therapeutic landscape, with Tyvaso accounting for roughly 30–35% of the inhalation-specific market share.

Potential Challenges

• Pipeline Competition: Oral and novel inhaled agents under development may erode Tyvaso’s market share.
• Pricing Pressures: Payers may negotiate discounts amid rising healthcare costs.
• Regulatory Hurdles: Any delays or restrictions in new indications could impact growth trajectory.

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Strategic Outlook

For stakeholders, leveraging ongoing clinical data, advocating for reimbursement inclusivity, and pursuing pipeline innovation are essential for capturing future value. Collaboration with academic institutions and real-world evidence collection will further validate Tyvaso's positioning, especially in combination therapies and expanded indications.

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Key Takeaways

• Clinical development remains robust, with upcoming Phase 3 data expected to influence label extensions and marketplace positioning.
• Market potential is substantial, driven by PAH prevalence, inhalation therapy benefits, and evolving treatment algorithms.
• Revenue projections indicate a compound annual growth rate of approximately 8% through 2030, reaching ~$1.2 billion globally.
• Competitive pressures vary, but Tyvaso retains an advantage owing to its inhalation route and established safety profile.
• Strategic focus on pipeline expansion, geographic penetration, and cost management will be critical to maximizing long-term growth.

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FAQs

1. What are the latest clinical trial results for Tyvaso?
   Recent Phase 3 trials highlight improvements in exercise capacity and quality of life, with continued safety across diverse patient populations. Data from the DURAND study is anticipated to further support broadened indications.

2. How does Tyvaso compare to other PAH therapies?
   Tyvaso offers inhaled prostacyclin delivery, balancing efficacy with ease of use and tolerability. It’s often preferred over intravenous options for patients seeking less invasive management, though it competes with oral agents like endothelin receptor antagonists.

3. What are the key factors influencing Tyvaso’s market growth?
   Factors include successful label expansion, increasing adoption in combination therapy, reimbursement policies, and global market penetration efforts.

4. Are there upcoming regulatory decisions that could impact Tyvaso?
   Positive outcomes from ongoing trials may lead to FDA and EMA label extensions, particularly for PPAP management and possibly other pulmonary hypertension forms.

5. What challenges might impede Tyvaso’s future growth?
   Competition from oral therapies, pricing reforms, and potential delays in clinical trial milestones could pose hurdles.

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Conclusion

Tyvaso remains a cornerstone in inhaled PAH therapy, with favorable clinical and market momentum. Strategic investments in clinical trial progression, market expansion, and pipeline innovation will underpin its growth prospects amid a competitive landscape. By continuously validating its efficacy and expanding indications, Tyvaso is poised to sustain its role as a vital treatment modality within the evolving pulmonary hypertension treatment paradigm.