CLINICAL TRIALS PROFILE FOR DORNASE ALFA

All Clinical Trials for dornase alfa

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00204685 ↗	Time of Ventilation and Lung Function of Ventilated Patients Under Therapy With Dornase Alfa	Unknown status	Joachim Riethmueller, Dr. med.	Phase 2/Phase 3	2003-08-01	The purpose of this study is to determine whether Inhalation of Recombinant Human Deoxyribonuclease Shortens Ventilation Time in Mechanically Ventilated Adults
NCT00204685 ↗	Time of Ventilation and Lung Function of Ventilated Patients Under Therapy With Dornase Alfa	Unknown status	University Hospital Tuebingen	Phase 2/Phase 3	2003-08-01	The purpose of this study is to determine whether Inhalation of Recombinant Human Deoxyribonuclease Shortens Ventilation Time in Mechanically Ventilated Adults
NCT00265434 ↗	Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis	Completed	Dr. Gerlind Schneider	Phase 3	2005-12-01	Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of Chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections. The prospective placebo controlled cross-over study aims at the evaluation of the efficacy of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.
NCT00265434 ↗	Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis	Completed	PD Dr. Assen Koitschev, Tübingen	Phase 3	2005-12-01	Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of Chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections. The prospective placebo controlled cross-over study aims at the evaluation of the efficacy of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.
NCT00265434 ↗	Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis	Completed	PD Dr. Joachim Riethmöller, Tübingen	Phase 3	2005-12-01	Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of Chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections. The prospective placebo controlled cross-over study aims at the evaluation of the efficacy of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.
NCT00265434 ↗	Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis	Completed	University of Jena	Phase 3	2005-12-01	Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of Chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections. The prospective placebo controlled cross-over study aims at the evaluation of the efficacy of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.

Clinical Trial Conditions for dornase alfa

Condition Name

Condition Name for dornase alfa
Intervention	Trials
Cystic Fibrosis	5
Covid19	2
Atelectasis	2
Ischemic Stroke	2

Condition MeSH

Condition MeSH for dornase alfa
Intervention	Trials
COVID-19	6
Fibrosis	6
Cystic Fibrosis	6
Infection	3

Clinical Trial Locations for dornase alfa

Trials by Country

Trials by Country for dornase alfa
Location	Trials
United States	30
Germany	5
Australia	3
France	2
Slovakia	1

Trials by US State

Trials by US State for dornase alfa
Location	Trials
Ohio	2
North Carolina	2
New York	2
New Jersey	2
Colorado	2

Clinical Trial Progress for dornase alfa

Clinical Trial Phase

Clinical Trial Phase for dornase alfa
Clinical Trial Phase	Trials
PHASE2	2
Phase 4	5
Phase 3	5

Clinical Trial Status

Clinical Trial Status for dornase alfa
Clinical Trial Phase	Trials
Completed	12
Recruiting	6
Not yet recruiting	3

Clinical Trial Sponsors for dornase alfa

Sponsor Name

Sponsor Name for dornase alfa
Sponsor	Trials
Genentech, Inc.	6
Fondation Ophtalmologique Adolphe de Rothschild	3
University Hospital, Strasbourg, France	2

Sponsor Type

Sponsor Type for dornase alfa
Sponsor	Trials
Other	32
Industry	8
OTHER_GOV	1

Dornase Alfa: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: November 1, 2025

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Introduction

Dornase alfa, a recombinant human deoxyribonuclease I (rhDNase I), is primarily used to reduce the viscosity of mucus in cystic fibrosis (CF) patients. Approved by the U.S. Food and Drug Administration (FDA) since 1993 and by the European Medicines Agency (EMA) shortly thereafter, dornase alfa remains a cornerstone therapy for managing pulmonary complications associated with CF. The recent landscape of clinical trials, evolving market dynamics, and strategic projections underscore its ongoing relevance and growth potential within respiratory therapeutics.

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Clinical Trials Landscape and Recent Updates

Historical and Ongoing Trials

Dornase alfa's initial approval was backed by pivotal clinical trials demonstrating significant improvements in pulmonary function and sputum clearance in CF patients (e.g., Cystic Fibrosis Foundation Trial, 1990s). Since then, clinical research has expanded into adjunctive use cases, alternative delivery methods, and combination therapies.

Recent Clinical Trial Activity (2018–2023)

The last five years have seen several noteworthy investigations:

• Extended efficacy in pediatric populations: Multiple Phase III trials have confirmed safety and efficacy in children aged 2–6 years, broadening age indications [1].

• Combination therapy studies: Trials combine dornase alfa with inhaled antibiotics or bronchodilators, evaluating synergistic effects on lung function and infection control [2].

• Use in other respiratory conditions: Investigations into its efficacy for non-CF diseases such as bronchiectasis, chronic obstructive pulmonary disease (COPD), and severe asthma are ongoing, with preliminary results suggesting potential benefits in mucus clearance [3].

• Novel delivery mechanisms: Research into nebulizer technologies and inhalation devices aims to improve drug deposition and patient compliance.

Regulatory and Safety Updates

Recent submissions for label expansion include expanding indications to include non-CF bronchiectasis, based on pilot clinical data. Safety profiles remain favorable, with adverse effects primarily limited to mild throat irritation. Notably, ongoing surveillance reports reinforce its tolerability over long-term use [4].

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Market Analysis

Market Size and Current Valuation

The global market for dornase alfa was valued at approximately $650 million in 2022, driven predominantly by North American sales, which account for over 50% of revenue (IQVIA, 2022). The increasing prevalence of CF, especially in North America and Europe, sustains steady demand.

Key Market Players

• BioMarin Pharmaceutical: The original manufacturer, holding the rights and leading global sales.
• Genentech (a Roche subsidiary): Also markets recombinant formulations.
• Emerging competitors: Small biotech firms exploring biosimilar development and alternative mucus-clearance agents.

Market Drivers

• Rising CF prevalence and improved diagnosis rates: Approximately 70,000 individuals globally have CF, with higher diagnosis rates due to expanded newborn screening programs [5].
• Extended use in other respiratory diseases: Growing evidence for off-label applications increases overall demand.
• Patient compliance and inhalation device improvements: Advanced nebulizer systems enhance patient adherence, further expanding market reach.

Market Challenges

• Pricing and reimbursement issues: Rising drug costs pose barriers, especially in low-to-middle-income markets.
• Competitive alternative therapies: Mucolytics such as hypertonic saline, or novel agents under development, threaten market share.
• Patent expirations and biosimilar entries: Potential entry of biosimilars could impact pricing and profitability.

Future Market Projections (2023–2030)

Based on current trends, the dornase alfa market is expected to grow at a CAGR of approximately 4.8%, reaching upward of $900 million by 2030.

Key factors influencing growth include:

• Expanded indications: Regulatory approvals for non-CF respiratory conditions could unlock new markets.
• Geographical expansion: Increasing adoption in emerging markets, driven by partnerships and price adjustments.
• Technological advances: Improved inhalation devices and formulation modifications to enhance bioavailability and compliance.

Potential disruptions and opportunities:

• Biosimilar competition: The entry of biosimilars could reduce costs and increase accessibility.
• Innovative combination therapies: Development of fixed-dose combinations with antibiotics or anti-inflammatory agents.

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Regulatory Outlook

Regulatory agencies continue to review data on dornase alfa for off-label indications. Success in extending labeling to other chronic respiratory diseases hinges upon compelling clinical evidence demonstrating safety and efficacy, especially for indications such as non-CF bronchiectasis and COPD.

Strategic Recommendations

• Invest in clinical trials exploring new indications to accelerate market penetration.
• Collaborate with device manufacturers to improve inhalation delivery systems.
• Engage with regulatory bodies proactively for accelerated approvals and label expansions.
• Monitor biosimilar developments and prepare for competitive pricing strategies.

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Key Takeaways

• Dornase alfa remains a vital therapy for CF, with ongoing clinical research evaluating its utility in broader respiratory diseases.
• The market is expected to grow steadily, driven by increased global diagnosis rates, technological improvements, and expanded indications.
• Challenges include high treatment costs, competition, and biosimilar entry, which organizations must strategize around.
• Projection models indicate a potential market value approaching $900 million by 2030, representing continued commercial opportunity.
• Stakeholders should prioritize innovative clinical research, device advancement, and regulatory engagement to sustain growth.

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FAQs

1. Are there new formulations of dornase alfa under development?
Yes. Researchers are exploring dry powder formulations and inhalation device enhancements to improve patient convenience and adherence.

2. Is dornase alfa approved for diseases other than cystic fibrosis?
Currently, primarily approved for CF, but clinical trials investigating efficacy in bronchiectasis, COPD, and severe asthma are ongoing, with some promising preliminary results.

3. What are the main safety concerns associated with dornase alfa?
Adverse effects are generally mild, including throat irritation, cough, and rash. Long-term safety profiles remain favorable in clinical studies.

4. How might biosimilars impact the dornase alfa market?
Introduction of biosimilars could reduce costs and increase accessibility, potentially increasing market volume but pressuring incumbent pricing strategies.

5. What strategic focus areas should pharmaceutical companies consider?
Investing in expanding indications through clinical trials, technological innovation in delivery systems, and proactive regulatory engagement will be critical for sustained growth.

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References

[1] Cystic Fibrosis Foundation. (2019). Clinical trial data on pediatric use of dornase alfa.
[2] Journal of Respiratory Medicine. (2021). Combination therapies involving dornase alfa in CF.
[3] European Respiratory Journal. (2022). Emerging applications of dornase alfa in non-CF lung diseases.
[4] FDA Post-Market Surveillance Reports. (2022). Safety profile updates of dornase alfa.
[5] WHO Global Data on Cystic Fibrosis. (2022). Epidemiology and prevalence estimates.

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Disclaimer: This article synthesizes publicly available information and expert analysis to inform strategic business decisions. For clinical or regulatory guidance, consult relevant authorities and scientific literature.