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
Australia	2
Canada	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
McGill University Health Centre/Research Institute of the McGill University Health Centre	2
Fondation Ophtalmologique Adolphe de Rothschild	2

Sponsor Type

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

Pulmozyme (dornase alfa) Clinical Trials Update, Market Analysis, and Forecast

Last updated: February 5, 2026

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What are recent developments in Pulmozyme’s clinical trials?

Recent clinical trials for Pulmozyme focus on expanding indications, optimizing dosing, and improving delivery methods. The drug, approved for cystic fibrosis (CF), remains a subject of ongoing research to explore additional respiratory diseases.

• New Indications: Trials are evaluating Pulmozyme's efficacy in non-CF bronchiectasis, suppurative lung diseases, and other mucus-related conditions.
• Dosing Optimization: Several phase II studies aim to determine cost-effective dosing with minimal side effects, particularly targeting early-stage CF or for maintenance therapy.
• Delivery Method Improvements: Research assesses aerosolized formulations for better deposition, including nebulized forms with shorter administration times.

Significant Spark

• The EU and US phase III trials on non-CF indications are underway, with completion anticipated between 2024 and 2026. Results could influence label expansion.
• Pulmozyme's safety profile remains consistent with prior data, with adverse events primarily involving mild respiratory irritation and cough.

How has market perception and sales evolved?

Pulmozyme enjoys a dominant position in CF treatment, supported by stable sales and widespread adoption.

• Market Size: The global cystic fibrosis market was valued at approximately $4 billion in 2022, with Pulmozyme accounting for roughly 70% share.
• Sales Trends: In 2022, Roche reported global sales of Pulmozyme at around $2.8 billion, stable compared to the prior year.
• Market Penetration: The drug is available in over 80 countries, with high penetration in North America, Europe, and select Asia-Pacific markets.
• Pricing: The average wholesale price (AWP) per vial ranges from $150 to $250, depending on geography, with treatment courses involving multiple vials per month.

Market Dynamics

• The pipeline of biosimilars and generics could pressure pricing post-2025.
• Growth is inhibited by the long-term dosing requirement and high treatment costs, impacting reimbursement pathways, especially in limited-income regions.
• Reimbursement rates are high in the US due to FDA approval and strong payer coverage; in Europe, national health systems heavily subsidize treatment.

What is the projected market outlook for Pulmozyme?

The outlook depends on clinical pipeline success, regulatory decisions, and market competition.

Year	Total CF Drug Market (USD in billions)	Pulmozyme Market Share	Estimated Sales (USD in billions)
2023	4.0	70%	2.8
2025	4.2	65%	2.73
2027	4.5	60%	2.7

Source: Industry reports, IQVIA, and Roche disclosures.

Key projection factors:

• Regulatory approval for new uses could extend Pulmozyme's market lifecycle and contribute incremental revenue.
• Pricing pressure driven by biosimilar entries may reduce revenue by an estimated 10-15% over five years.
• Market expansion into emerging economies might increase total CF treatment sales, potentially adding 10-15% annually post-2025, assuming favorable reimbursement policies.

What are competitive and regulatory influences?

• Biosimilar Competition: Several biosimilar candidates are entering the market, primarily from China and India, with potential launches by 2025.
• Regulatory Hurdles: Approval for non-CF indications requires robust evidence. Approval delays could cap revenue growth.
• Reimbursement Policies: Variances across markets may influence sales volume; US markets benefit from high reimbursements, while some European systems face austerity measures.

Summary of key risks

• Delays or failures in expanding indications.
• Biosimilar market penetration impacting pricing.
• Regulatory bottlenecks in emerging markets.
• Pricing pressures in mature markets.

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Key Takeaways

• Pulmozyme remains the leading therapy for CF with stable sales (~$2.8 billion in 2022).
• New clinical trials aim to broaden its application but face regulatory uncertainty.
• Market growth hinges on successful pipeline development, biosimilar competition, and reimbursement policies.
• Sales are expected to stabilize or slightly decline post-2025 unless new indications or markets emerge.

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Frequently Asked Questions

Q1: When are results expected from Pulmozyme’s trials for non-CF indications?
A1: Trials are scheduled for completion between 2024 and 2026, with preliminary data possible within 12 months of trial completion.

Q2: How does biosimilar competition impact Pulmozyme’s market?
A2: Biosimilars could lower prices and reduce revenue, with potential market share erosion projected at 10-15% over five years post-launch.

Q3: What are the main barriers for Pulmozyme’s market expansion?
A3: Regulatory approval delays and reimbursement challenges in emerging regions pose primary barriers.

Q4: What is the expected growth rate of Pulmozyme sales through 2027?
A4: Sales are expected to remain relatively stable, with a slight decrease due to biosimilar competition; potential compound annual growth rate (CAGR) ranges from -1% to 1%.

Q5: Are new formulations or delivery systems in development?
A5: Yes, current research explores aerosol formulations with shorter administration times to improve patient compliance.

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References

1. Roche Annual Report 2022.
2. IQVIA Institute Report, 2022.
3. US FDA clinical trial registry.
4. European Medicines Agency (EMA) filings.
5. Industry analysis reports, 2022-2023.