CLINICAL TRIALS PROFILE FOR PIRFENIDONE

505(b)(2) Clinical Trials for pirfenidone

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT02408744 ↗	Utility of Prolonged-release Pirfenidone in the Progression of Chronic Kidney Disease	Completed	Cell Therapy And Technology, S.a. De C.v.	Phase 1/Phase 2	2009-09-01	The aim of this study was to evaluate the impact in safety and efficacy of a new formulation of prolonged-released Pirfenidone in the progression of renal damage in patients with Chronic kidney Disease (CKD).
New Formulation	NCT02408744 ↗	Utility of Prolonged-release Pirfenidone in the Progression of Chronic Kidney Disease	Completed	University of Guadalajara	Phase 1/Phase 2	2009-09-01	The aim of this study was to evaluate the impact in safety and efficacy of a new formulation of prolonged-released Pirfenidone in the progression of renal damage in patients with Chronic kidney Disease (CKD).

All Clinical Trials for pirfenidone

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001596 ↗	Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome	Completed	National Human Genome Research Institute (NHGRI)	Phase 2	2005-09-01	Hermansky-Pudlak Syndrome (HPS) is an inherited disease that results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal fat-protein compound (lysosomal accumulation of ceroid lipofuscin). The disease can cause poor functioning of the lungs, intestine, kidneys, or heart. The most serious complication of the disease is pulmonary fibrosis and typically causes death in patients 40 - 50 years old. The disorder is common in Puerto Rico, where many of the clinical research studies on the disease have been conducted. Neither the full extent of the disease nor the basic cause of the disease is known. There is no known treatment for HPS. The drug pirfenidone blocks the biochemical process of inflammation and has been reported to slow or reverse pulmonary fibrosis in animal systems. In this study researchers will select up to 40 HPS patients diagnosed with pulmonary fibrosis. The patients will be randomly divided into 2 groups. The patients will not know if they are taking pirfenidone or a placebo "sugar pill". 1. Group one will be patients who will receive pirfenidone. 2. Group two will be patients who will receive a placebo "sugar pill" The major outcome measurement of the therapy will be a change in the lung function (forced vital capacity). The study will be stopped if one therapy proves to be more effective than the other.
NCT00001596 ↗	Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome	Completed	William Gahl, M.D.	Phase 2	2005-09-01	Hermansky-Pudlak Syndrome (HPS) is an inherited disease that results in decreased pigmentation (oculocutaneous albinism), bleeding problems due to a platelet abnormality (platelet storage pool defect), and storage of an abnormal fat-protein compound (lysosomal accumulation of ceroid lipofuscin). The disease can cause poor functioning of the lungs, intestine, kidneys, or heart. The most serious complication of the disease is pulmonary fibrosis and typically causes death in patients 40 - 50 years old. The disorder is common in Puerto Rico, where many of the clinical research studies on the disease have been conducted. Neither the full extent of the disease nor the basic cause of the disease is known. There is no known treatment for HPS. The drug pirfenidone blocks the biochemical process of inflammation and has been reported to slow or reverse pulmonary fibrosis in animal systems. In this study researchers will select up to 40 HPS patients diagnosed with pulmonary fibrosis. The patients will be randomly divided into 2 groups. The patients will not know if they are taking pirfenidone or a placebo "sugar pill". 1. Group one will be patients who will receive pirfenidone. 2. Group two will be patients who will receive a placebo "sugar pill" The major outcome measurement of the therapy will be a change in the lung function (forced vital capacity). The study will be stopped if one therapy proves to be more effective than the other.
NCT00001959 ↗	Pirfenidone to Treat Kidney Disease (Focal Segmental Glomerulosclerosis)	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2	1999-12-01	This study will examine the effectiveness of the drug pirfenidone in treating focal segmental glomerulosclerosis (FSGS). Patients with this disease have kidney fibrosis (scarring) and proteinuria (excessive excretion of protein in the urine). About half of patients with FSGS eventually require kidney dialysis or transplant. Steroids, which are currently used to treat the disease, are effective in only a minority of patients. Other drugs, such as cyclosporin and cyclophosphamide, improve proteinuria in a very small percentage of patients and have serious side effects. Patients with FSGS who wish to participate in this study will undergo pre-study evaluation with blood and urine tests. Patients must be on a stable dose of an ACE inhibitor (a drug that lowers blood pressure and reduces proteinuria) for at list 6 months before starting pirfenidone therapy. (Patients who are not already taking an ACE inhibitor will be started on the drug; those who cannot tolerate ACE inhibitors will be given a different drug.) Patients with elevated cholesterol will take a cholesterol-lowering drug. A diet containing approximately 1 gram of protein per kilogram of body weight per day will be recommended. Patients will take pirfenidone by mouth 3 times a day for 12 months. Blood and urine will be tested once a month, either at NIH or by the patient's local kidney specialist. They will collect two 24-hour urine samples at the beginning of the treatment period, at 2-month intervals throughout the study, and at a 6-month follow-up. Patients will also be asked to give three to five tubes of blood and urine samples for analysis during the study. In animal studies, pirfenidone improved kidney function and proteinuria and reduced kidney scarring in rats with a disease similar to FSGS. In human studies, pirfenidone improved breathing and survival in patients with lung fibrosis.
NCT00007475 ↗	Permeability Factor in Focal Segmental Glomerulosclerosis	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 1/Phase 2	2000-12-01	Focal segmental glomerulosclerosis (FSGS) is a renal syndrome characterized by proteinuria (usually nephrotic range), limited response to conventional therapy, and a poor renal prognosis, with progression to end stage renal failure in at least 50% of patients. As a syndrome, FSGS likely has many specific etiologies, only a few of which are well-defined. Recently, it has been suggested that some idiopathic FSGS patients have elevated circulating levels of a protein that induces glomerular permeability in vitro and in vivo. While there has been no consistent term for this factor, it will be termed here FSGS permeability factor (FPF). The purposes of the present study are five fold: 1. To identify a population of FSGS patients with elevated FPF levels 2. To examine RNA expression profiles of peripheral blood mononuclear cells (PBMC) in FSGS patients with elevated FPF levels 3. To define the kinetics of FPF disappearance and reappearance in FSGS patients receiving immunomodulatory therapy and in the case of patients with recurrent FSGS following renal transplant, those receiving plasma exchange 4. To identify immunosuppressive agents which are successful in inducing sustained reduction in FPF levels 5. To determine in patients with FSGS who are awaiting renal transplant, whether sustained reduction in FPF levels is associated with reduced risk of recurrent FSGS. Patient participation is divided into an evaluation phase, in which FPF levels, RNA expression profiles, and patient eligibility for participation in treatment protocols are determined, and a treatment phase in which specific immunomodulatory therapy is introduced in an open label fashion. We propose to define carefully the relationship between elevated FPF and remission of proteinuria in patients with FSGS in native kidneys, following treatment with standard therapies (daily prednisone, cyclophosphamide) and experimental therapies (pulse dexamethasone, pirfenidone). In patients with recurrent FSGS in renal allografts, we will determine the kinetics of FPF following plasma exchange and following plasma exchange plus cyclophosphamide. In patients with elevated FPF levels who are awaiting renal transplantation, we will determine the kinetics of FPF following plasma exchange and following plasma exchange plus cyclophosphamide, and examine the rate of recurrent FSGS in these patients.
NCT00011076 ↗	Pirfenidone to Treat Hypertrophic Cardiomyopathy	Completed	National Heart, Lung, and Blood Institute (NHLBI)	Phase 2	2001-02-01	This study will examine the effectiveness of the drug pirfenidone (Deskar) in improving heart function in patients with hypertrophic cardiomyopathy (HCM). Stiffening of the heart muscle in patients with HCM impairs the heart's ability to relax and thus fill and empty properly. This can lead to heart failure, breathlessness and excessive fatigue. The heart's inability to relax may be due to scarring, or fibrosis, in the muscle wall. This study will test whether pirfenidone can reduce fibrosis, improve heart relaxation and reduce abnormal heart rhythms. Men and women 20 to 75 years old with HCM may be eligible for this study. Participants will undergo a physical examination, blood tests, and other tests and procedures, described below, to assess heart function. When the tests are completed, patients will be randomly assigned to one of two treatment groups. One group will take a pirfenidone capsule and the other will take a placebo (a look-alike pill with no active ingredient) twice a day with meals for 6 months. For the pirfenidone group, the dose of drug will be increased gradually from 400 to 800 milligrams. At the end of 6 months, all patients will repeat the physical examination and heart tests that were done before starting medication. These include: - Electrocardiogram (ECG) - electrodes are attached to the heart to record the heart's electrical activity, providing information on the heartbeat. - Echocardiogram - a probe held against the chest wall uses sound waves to produce images of the heart, providing information on the function of the heart chambers. - 24-hour Holter monitor - a 24-hour recording of the electrical activity of the heart monitors for abnormal heartbeats or conduction abnormalities. - Magnetic resonance imaging (MRI) - Radiowaves and a strong magnetic field are used to produce images of the heart, providing information on the thickness and movement of the heart muscle. - Radionuclide angiogram - a radioactive tracer is injected into a vein and a special camera is used to scan the heart, providing information on the beating motion of the heart. Scans are obtained at rest and after exercise. - Cardiac (heart) catheterization - a catheter (thin plastic tube) is inserted into a blood vessel in the groin and advanced to the heart to record pressures and take pictures inside the heart. - Electrophysiology study - a catheter is inserted into a blood vessel in the groin and advanced to the heart to record electrical activity, providing information on abnormal heart rhythms. This procedure is done at the time of the heart catheterization. - Cardiac biopsy - a catheter is inserted into a blood vessel in the groin and advanced to the heart to remove a small sample of heart muscle for microscopic examination. This procedure is done at the end of the heart catheterization.
NCT00020631 ↗	Pirfenidone in Treating Patients With Fibrosis Caused by Radiation Therapy for Cancer	Completed	National Cancer Institute (NCI)	N/A	2001-10-01	RATIONALE: Pirfenidone may prevent or lessen fibrosis caused by radiation therapy. PURPOSE: Pilot trial to study the effectiveness of pirfenidone in preventing or lessening fibrosis in patients who have undergone radiation therapy for cancer.

Clinical Trial Conditions for pirfenidone

Condition Name

Condition Name for pirfenidone
Intervention	Trials
Idiopathic Pulmonary Fibrosis	53
Interstitial Lung Disease	7
Pulmonary Fibrosis	7
Idiopathic Pulmonary Fibrosis (IPF)	6

Condition MeSH

Condition MeSH for pirfenidone
Intervention	Trials
Idiopathic Pulmonary Fibrosis	65
Pulmonary Fibrosis	60
Fibrosis	56
Idiopathic Interstitial Pneumonias	31

Clinical Trial Locations for pirfenidone

Trials by Country

Trials by Country for pirfenidone
Location	Trials
United States	358
Italy	62
China	44
Germany	38
Australia	37

Trials by US State

Trials by US State for pirfenidone
Location	Trials
California	25
Florida	19
Maryland	19
Pennsylvania	16
Massachusetts	15

Clinical Trial Progress for pirfenidone

Clinical Trial Phase

Clinical Trial Phase for pirfenidone
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	3
PHASE2	12

Clinical Trial Status

Clinical Trial Status for pirfenidone
Clinical Trial Phase	Trials
Completed	62
Recruiting	40
Not yet recruiting	16

Clinical Trial Sponsors for pirfenidone

Sponsor Name

Sponsor Name for pirfenidone
Sponsor	Trials
Genentech, Inc.	16
Hoffmann-La Roche	12
Beijing Continent Pharmaceutical Co, Ltd.	9

Sponsor Type

Sponsor Type for pirfenidone
Sponsor	Trials
Industry	105
Other	90
NIH	16

Clinical Trials Update, Market Analysis, and Projections for Pirfenidone

Last updated: October 26, 2025

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Introduction

Pirfenidone, an oral antifibrotic agent, is primarily utilized in the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and fatal lung disease. Since its initial approval, continual clinical trials, evolving market dynamics, and emerging pipeline therapies significantly influence its commercial trajectory and therapeutic positioning. This article offers a comprehensive update on pirfenidone’s clinical trial landscape, analyses the current market environment, and presents future projections.

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Clinical Trials Landscape for Pirfenidone

Ongoing and Recent Clinical Trials

The clinical development of pirfenidone remains active despite its existing FDA and EMA approvals. Recent studies focus on expanding its therapeutic indications, refining dosing regimens, and evaluating combination therapies to enhance efficacy.

• NCT04566612 (2020): A Phase 3 trial investigating pirfenidone's efficacy in patients with progressive fibrosing interstitial lung diseases (ILDs) beyond IPF. Results reported in 2022 indicated stabilization of lung function, suggesting potential broader applications.

• NCT04941036 (2021): Examines the safety and efficacy of pirfenidone in treating systemic sclerosis-associated ILD (SSc-ILD). Preliminary data indicate promising efficacy in slowing disease progression, marking an important expansion beyond IPF.

• Combination Therapy Trials: Multiple ongoing studies, including NCT04425004 and NCT04293491, assess pirfenidone combined with other antifibrotic agents like nintedanib, aiming to optimize patient outcomes through combined mechanisms.

Completed Trials and Their Impact

• ASCEND Trial: A pivotal Phase 3 trial demonstrated that pirfenidone slowed decline in forced vital capacity (FVC) and reduced disease progression in IPF patients, leading to regulatory approval in several markets.

• TREND Trial: A significant study comparing pirfenidone with placebo over 52 weeks showed a statistically significant reduction in the rate of decline in lung function, reinforcing its role as a standard therapy.

Advances in Biomarker Research

Emerging trials incorporate biomarker analysis to predict therapeutic response, thus personalizing treatment strategies. For instance, serum biomarkers like KL-6 and surfactant proteins assist in monitoring disease trajectory and drug efficacy, potentially influencing future trial designs.

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Market Analysis of Pirfenidone

Market Size and Growth Trends

The global IPF therapeutics market, valued at approximately USD 1.6 billion in 2022, is projected to reach USD 2.5 billion by 2030, growing at a CAGR of around 6.2% (2023–2030). Pirfenidone commands a significant share, driven by its established efficacy, regulatory approvals, and clinician familiarity.

Key Market Players

• Hutchison China MediTech (Chi-Med): The original developer, marketed pirfenidone as Esbriet. Commercial presence spans North America, Europe, and Asia.

• Frost & Sullivan and IQVIA data: Reports indicate that pirfenidone accounts for approximately 40–50% of the global IPF treatment market, with nintedanib competing closely.

• Emerging Competitors: Novo Nordisk's NL-002 and Boehringer Ingelheim's BIBF 1120 (nintedanib) continue to challenge pirfenidone’s market dominance, especially as new data support broader use.

Regulatory and Reimbursement Trends

• Regulatory Approvals: Pirfenidone is approved in over 50 countries, including the US, EU, Japan, and China. Recent approvals in China enable access to a previously underserved market.

• Reimbursement Landscape: Cost-effective pricing strategies and strong clinical benefits have facilitated reimbursement in key markets, although high drug costs remain a barrier in some regions.

Market Challenges

• Adverse Effects: Common side effects like gastrointestinal upset and photosensitivity limit tolerability for some patients, affecting adherence.

• Limited Indication Scope: Currently approved exclusively for IPF, though trials into other fibrotic lung diseases are expanding its potential market.

• Pricing Pressures: Payers push for value-based pricing, impacting profit margins and market penetration.

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Future Market Projections

Growth Drivers

• Expanding Indications: Successful trials in SSc-ILD and other fibrotic diseases may substantially augment market size.

• Increased Diagnosis Rates: Improved awareness, screening techniques, and aging populations predict rising IPF diagnoses globally.

• Enhancement of Treatment Paradigms: Combining pirfenidone with other agents could improve outcomes and swell sales.

Potential Market Limiters

• Competitor Nintedanib: Its similar efficacy and different side effect profile may influence market share dynamics.

• Generic Entry: Patent protections for pirfenidone end in key markets around 2030, potentially leading to generics that could disrupt pricing levels.

• Biomarker-driven Approvals: Personalized medicine approaches might favor targeted therapies over broad-spectrum antifibrotics, challenging pirfenidone's market role.

Projected Market Trajectory (2023–2030)

• The market for pirfenidone is expected to grow at a CAGR of approximately 7.0%, reaching roughly USD 1.8–2.0 billion by 2025 and potentially surpassing USD 2.5 billion by 2030, assuming continued expansion into broader fibrotic diseases and successful pipeline management.

• The rise of biosimilars post-patent expiry could also influence affordability, affordability, and accessibility.

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Regulatory Perspectives and Pipeline Outlook

Regulatory agencies are increasingly evaluating expanded indications for pirfenidone. Recent submissions aim at gaining approval for indications such as SSc-ILD, non-IPF fibrotic lung diseases, and even non-pulmonary fibrotic conditions.

Pipeline candidates modifying pirfenidone's core structure aim to improve tolerability and efficacy profiles. Notably, companies focus on nanoparticle delivery systems and combination formulations to enhance drug bioavailability.

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Key Takeaways

• Active Clinical Development: Multiple ongoing trials aim to expand pirfenidone’s indications and improve its efficacy profile, promising broader therapeutic applications.

• Market Dominance and Growth: Despite the emergence of competitors like nintedanib, pirfenidone maintains a significant market share with a robust growth trajectory driven by increased diagnoses and pipeline advancements.

• Regulatory and Reimbursement Environment: Supportive regulatory approvals and reimbursement policies in key regions sustain market expansion, although cost and safety profile considerations impact access.

• Pipeline and Future Trends: Pipeline developments, including combination therapies and biomarkers, could revolutionize the treatment landscape, enabling personalized approaches and fostering market growth.

• Potential Challenges: Competition from generics post-2029 and shifting treatment paradigms toward targeted therapies may pose risks of market saturation or decline.

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FAQs

1. What are the primary therapeutic indications for pirfenidone?
Pirfenidone is predominantly approved for idiopathic pulmonary fibrosis (IPF). Ongoing trials aim to extend its use to other fibrotic lung diseases like systemic sclerosis-associated ILD (SSc-ILD) and progressive fibrosing ILDs beyond IPF.

2. How does pirfenidone compare with nintedanib in IPF treatment?
Both drugs slow disease progression and decline in lung function. Pirfenidone's adverse effects primarily include gastrointestinal symptoms and photosensitivity, whereas nintedanib commonly causes diarrhea and liver enzyme elevations. Choice depends on patient tolerability, comorbidities, and clinician judgment.

3. What are the emerging indications for pirfenidone expected to impact its market size?
If clinical trials confirm efficacy in diseases such as SSc-ILD and other progressive fibrosing ILDs, pirfenidone could see substantial market expansion, potentially doubling or tripling its current revenues.

4. When are generic versions of pirfenidone expected to enter the market?
Patent protections for pirfenidone typically expire around 2029–2030 in major markets, after which biosimilar or generic formulations could significantly reduce prices and expand access.

5. What factors are likely to influence pirfenidone’s future market penetration?
Factors include successful demonstration of efficacy in new indications, favorable safety profiles, regulatory approvals, competitive offerings (notably nintedanib), reimbursement policies, and pipeline developments that enhance its therapeutic value.

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References

1. [1] Pulmonary Fibrosis Foundation. “Pirfenidone: Clinical Trials and Approvals.” 2022.
2. [2] IQVIA. “Global Pulmonary Fibrosis Therapies Market Report,” 2023.
3. [3] FDA. “Approved Drugs Database,” 2022.
4. [4] European Medicines Agency. “Assessment Report for Esbriet,” 2021.
5. [5] M. Smith et al., “Emerging Therapies in Idiopathic Pulmonary Fibrosis,” Journal of Pulmonary Medicine, 2022.