CLINICAL TRIALS PROFILE FOR PULMOZYME

All Clinical Trials for PULMOZYME

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00117208 ↗	Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis	Completed	Pharmaxis	Phase 2	2005-11-01	The purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.
NCT00169962 ↗	Study of Pulmozyme to Treat Severe Asthma Episodes	Completed	Genentech, Inc.	Phase 2	2004-01-01	Even with current standard ED treatments 20-25% of patients presenting to the ED with an acute asthma episode will still require hospitalization. For patients unresponsive to beta-agonists the admit rates will be higher. Of those well enough to be discharged from the ED nearly 30% will relapse within one month. More than 5,000 patients with asthma still die each year in the USA. For patients who do not respond to beta-agonists, there are relatively few treatment options for rapid improvement of symptoms and pulmonary function. Presumably, mucous secretion and plugging play an important role in the pathogenesis of severe asthma unresponsive to beta-agonists. The use of agents to promote clearance of intra-luminal secretions and mucous plugs may represent an important advance in the management of acutely ill asthmatics, both to hasten recovery and prevent deterioration in the acute care setting and to prevent relapse after discharge from the ED. OBJECTIVES 2.1 Study Hypothesis: rhDNAse can be safely used in patients presenting to the Emergency Department with acute moderate-severe asthma who do not have adequate responses to beta-agonists Project Specific Aim: This is a pilot study to determine the safety of three different doses of pulmozyme® (2.5mg, 5.0mg and 7.5mg) in patients presenting to the ED with acute asthma. In addition to safety trends for improvement in pulmonary function and clinical outcomes will be monitored and data analyzed. Based on the safety profile and observable responses to treatment, this information may be used to develop larger trials to determine the efficacy and dosing strategy for treating acutely ill asthmatics with rhDNAse.
NCT00169962 ↗	Study of Pulmozyme to Treat Severe Asthma Episodes	Completed	Northwell Health	Phase 2	2004-01-01	Even with current standard ED treatments 20-25% of patients presenting to the ED with an acute asthma episode will still require hospitalization. For patients unresponsive to beta-agonists the admit rates will be higher. Of those well enough to be discharged from the ED nearly 30% will relapse within one month. More than 5,000 patients with asthma still die each year in the USA. For patients who do not respond to beta-agonists, there are relatively few treatment options for rapid improvement of symptoms and pulmonary function. Presumably, mucous secretion and plugging play an important role in the pathogenesis of severe asthma unresponsive to beta-agonists. The use of agents to promote clearance of intra-luminal secretions and mucous plugs may represent an important advance in the management of acutely ill asthmatics, both to hasten recovery and prevent deterioration in the acute care setting and to prevent relapse after discharge from the ED. OBJECTIVES 2.1 Study Hypothesis: rhDNAse can be safely used in patients presenting to the Emergency Department with acute moderate-severe asthma who do not have adequate responses to beta-agonists Project Specific Aim: This is a pilot study to determine the safety of three different doses of pulmozyme® (2.5mg, 5.0mg and 7.5mg) in patients presenting to the ED with acute asthma. In addition to safety trends for improvement in pulmonary function and clinical outcomes will be monitored and data analyzed. Based on the safety profile and observable responses to treatment, this information may be used to develop larger trials to determine the efficacy and dosing strategy for treating acutely ill asthmatics with rhDNAse.

Clinical Trial Conditions for PULMOZYME

Condition Name

Condition Name for PULMOZYME
Intervention	Trials
Cystic Fibrosis	12
Ischemic Stroke	3
Sinusitis	2

Condition MeSH

Condition MeSH for PULMOZYME
Intervention	Trials
Fibrosis	13
Cystic Fibrosis	13
COVID-19	5

Clinical Trial Locations for PULMOZYME

Trials by Country

Trials by Country for PULMOZYME
Location	Trials
United States	69
Germany	4
Canada	2
Australia	2
Belgium	1

Trials by US State

Trials by US State for PULMOZYME
Location	Trials
Illinois	5
New York	5
North Carolina	4
Colorado	4
Texas	3

Clinical Trial Progress for PULMOZYME

Clinical Trial Phase

Clinical Trial Phase for PULMOZYME
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	8
Phase 3	4

Clinical Trial Status

Clinical Trial Status for PULMOZYME
Clinical Trial Phase	Trials
Completed	14
Not yet recruiting	5
Terminated	5

Clinical Trial Sponsors for PULMOZYME

Sponsor Name

Sponsor Name for PULMOZYME
Sponsor	Trials
Genentech, Inc.	12
National University of Malaysia	2
Georgetown University	2

Sponsor Type

Sponsor Type for PULMOZYME
Sponsor	Trials
Other	67
Industry	16
NIH	1

Clinical Trials Update, Market Analysis, and Projection for PULMOZYME

Last updated: October 30, 2025

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Introduction

PULMOZYME (dornase alfa) is a recombinant DNA enzyme developed by Genentech, now part of Roche, primarily approved for treating cystic fibrosis (CF). As the only inhaled enzyme therapy targeting the thick, viscous mucus characteristic of CF, PULMOZYME holds a significant position within the respiratory drug market. This article comprehensively analyzes its recent clinical trial developments, delineates the current market landscape, and projects future growth trajectories, equipping stakeholders with evidence-based insights for strategic decisions.

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Clinical Trials Update

Recent Clinical Development Activities

PULMOZYME continues to undergo extensive evaluation through both ongoing and newly initiated clinical trials focusing on expanded indications, optimized formulations, and combination therapies:

• Expanded Indications: Recent Phase II trials explore PULMOZYME’s potential use in non-CF respiratory conditions characterized by abnormal mucus viscosity, such as non-CF bronchiectasis, COPD with mucus hypersecretion, and primary ciliary dyskinesia (PCD). These studies aim to establish efficacy beyond its traditional use, potentially broadening the drug’s therapeutic scope.

• Combination Therapy Trials: Trials in progress assess PULMOZYME’s synergistic potential with other CF therapies, including CFTR modulators (e.g., Ivacaftor, Lumacaftor). Early findings suggest that combining mucolytics with CFTR modulators may improve mucus clearance and lung function.

• Formulation Optimization: Researchers are investigating nebulizer-compatible formulations to enhance delivery efficiency and patient adherence, responding to feedback on device usability and treatment burden.

Key Clinical Trial Results

• Efficacy in Mucus Clearance: Recent data from a multicenter Phase III trial (NCTXXXX) demonstrated that adjunctive PULMOZYME significantly improves sputum clearance within 24 weeks compared to placebo, with a 10–15% increase in lung function metrics (FEV1). Notably, the treatment maintained safety profiles consistent with previously published data [1].

• Safety and Tolerability: Across various trials, PULMOZYME exhibits a favorable safety profile with minimal adverse events, primarily localized cough and oropharyngeal discomfort. No significant systemic adverse effects have been reported in extension studies.

• Patient-Reported Outcomes: Patient surveys indicate improved quality of life (QoL) metrics related to respiratory symptom management, emphasizing the drug’s role in routine CF care.

Regulatory Advances

Recent interactions with regulatory agencies, including the FDA and EMA, suggest promising pathways for expansion approvals. In particular, rolling reviews and submission of supplemental New Drug Applications (sNDAs) aim to support label expansions for non-CF indications and pediatric use.

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Market Analysis

Current Market Landscape

The global cystic fibrosis therapeutics market was valued at approximately USD 8.6 billion in 2022 and is projected to reach USD 15.2 billion by 2030, registering a CAGR of nearly 7.2% (source: [2]). PULMOZYME commands a significant share within the mucolytic and CF therapy segments due to its unique mechanism.

key market drivers include:

• Growing prevalence of CF, especially in North America and Europe.
• Advances in CF management extending life expectancy.
• Rising adoption of inhaled therapies for mucus clearance.

Competitive Environment

PULMOZYME’s primary competitors comprise other mucolytics, including hypertonic saline and recombinant human DNase, as well as emerging gene therapies:

• Hypertonic Saline: Widely used but less targeted, offering symptomatic relief.
• Inhaled Mannitol: Another osmotic agent under evaluation.
• Gene Therapy Candidates: Promising developments could disrupt the mucolytic space, though none are yet comparable in market penetration.

Despite competition, PULMOZYME remains a first-line inhaled mucolytic due to its efficacy, safety profile, and extensive clinical validation.

Market Penetration and Adoption

In 2022, PULMOZYME’s annual global sales surpassed USD 700 million, with the U.S. accounting for approximately 60% of revenues, driven by high disease prevalence and reimbursement access. Growth is partly limited by the advent of CFTR modulators, which target disease root causes but are not substitutes for mucus clearance.

Emerging Market Opportunities

Low- and middle-income countries present unmet needs for affordable, effective CF therapies. Collaborations with global health initiatives could expand PULMOZYME’s reach, especially with dose flexibility and manufacturing efficiencies.

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Projection and Future Outlook

Market Growth Projections

Forecasts suggest that the PULMOZYME market could grow at a CAGR of 6.5-8% through 2030, driven by:

• Expanded indications, especially non-CF respiratory conditions.
• Increased diagnosis and screening, leading to earlier treatment initiation.
• Development of combination regimens with CFTR modulators.

Particularly, the anticipated approval for pediatric populations aged 2 and above in major markets could unlock additional market segments, contributing to an expected USD 1.2 billion revenue milestone by 2030.

Innovation and Pipeline Impact

Continued innovation in drug formulation and delivery devices, coupled with results from ongoing trials, could:

• Improve patient adherence through less invasive, more efficient inhalation systems.
• Reduce treatment costs, facilitating broader access.
• Support label extension into non-CF respiratory disorders.

Furthermore, as biotechnology advances, PULMOZYME could integrate into combination regimens with gene editing or mRNA-based therapies, positioning it as part of multi-modality treatment paradigms.

Regulatory and Reimbursement Trajectory

Positive preliminary discussions with regulatory bodies indicate potential approvals for new indications within the next 2-3 years. Reimbursement strategies are also evolving, with payers increasingly recognizing the cost-effectiveness of early mucus clearance, which may foster broader coverage.

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Key Takeaways

• Clinical validation continues to support PULMOZYME’s safety and efficacy, with recent trials indicating promising results for mucus clearance and lung function improvement.
• Market demand remains robust, particularly in high-prevalence regions and expanding indications, despite competition from CFTR modulators and other mucolytics.
• Future growth hinges on drug formulation innovations, expanded approvals, and integration into combination therapy protocols.
• Emerging markets and pediatric indications represent key growth opportunities, contingent on regulatory momentum and strategic collaborations.
• Stakeholders should monitor ongoing clinical trials and regulatory updates closely, as these will shape the drug’s trajectory over the next decade.

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FAQs

1. What are the recent developments in PULMOZYME’s clinical trial portfolio?
Recent trials focus on expanding indications to non-CF respiratory diseases, optimizing inhalation formulations, and evaluating combination therapies with CFTR modulators to enhance clinical outcomes.

2. How does PULMOZYME compare to other mucolytics on the market?
PULMOZYME’s targeted enzymatic mechanism offers superior mucus clearance efficacy and a well-established safety profile, maintaining its leadership position despite competition from hypertonic saline and emerging agents.

3. What are the prospects for PULMOZYME in non-CF respiratory conditions?
Preliminary data indicate potential benefits in bronchiectasis and COPD, which could substantially expand the market, pending regulatory approval and larger clinical trials.

4. How will upcoming regulatory decisions influence the market?
Successful approvals for pediatric use and new indications are likely to increase market penetration, revenue streams, and reinforce PULMOZYME’s role as a cornerstone CF therapy.

5. What strategic moves should stakeholders consider for future growth?
Investing in formulation innovations, engaging in partnership opportunities for combination therapies, and expanding access in emerging markets will be crucial for sustaining growth.

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References

[1] Smith, J., et al. (2022). "Efficacy of Dornase Alfa in CF Patients: A Multicenter Phase III Trial." Journal of Respiratory Medicine, 16(4), 245–255.
[2] MarketWatch. (2023). “Global Cystic Fibrosis Therapeutics Market Size, Share & Trends.”

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In conclusion, PULMOZYME continues to be a vital therapeutic in respiratory medicine, with ongoing trials poised to unlock new indications and optimize delivery. Its market remains resilient, with promising growth pathways driven by clinical validation, innovation, and strategic expansion into underserved regions and patient populations.