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.
NCT00179998 ↗	Effectiveness of Pulmozyme in Infants With Cystic Fibrosis	Completed	Genentech, Inc.	Phase 2	2005-01-01	This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
NCT00179998 ↗	Effectiveness of Pulmozyme in Infants With Cystic Fibrosis	Completed	Nationwide Children's Hospital	Phase 2	2005-01-01	This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
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.

Clinical Trial Conditions for PULMOZYME

Condition Name

Condition Name for PULMOZYME
Intervention	Trials
Cystic Fibrosis	12
Ischemic Stroke	3
Sinusitis	2
COVID-19	2

Condition MeSH

Condition MeSH for PULMOZYME
Intervention	Trials
Fibrosis	13
Cystic Fibrosis	13
COVID-19	5
Infections	4

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
Pennsylvania	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
Georgetown University	2
University of Jena	2

Sponsor Type

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

PULMOZYME (dornase alfa): Clinical Trials Update, Market Analysis, and Projection

Last updated: May 7, 2026

What is PULMOZYME and where is it positioned commercially?

PULMOZYME is dornase alfa (rhDNase), an inhaled recombinant human DNase I indicated to improve lung function in people with cystic fibrosis (CF). The product’s commercial footprint is tied to CF epidemiology, guideline adoption, payer coverage, and competitive dynamics among CF therapies (including CFTR modulators).

Key product facts

• Active ingredient: dornase alfa (rhDNase)
• Route/formulation: inhaled
• Core indication: cystic fibrosis (improves lung function)

Commercial implication

• As CFTR modulators expanded, the role of DNase alfa shifts from “foundational CF anti-inflammation/mucus strategy” to a complementary therapy used based on residual disease burden, pulmonary phenotype, and payer policies.

What is the clinical trials landscape for PULMOZYME?

A complete, up-to-date “clinical trials update” requires trial-level records (NCT IDs, status, endpoints, and dates). Without verified access to the latest registry snapshots and sponsor publications, a definitive trial status summary cannot be produced without risking accuracy.

Therefore, no trial-by-trial update is provided here.

How big is the addressable market for PULMOZYME?

Market drivers

• CF prevalence and treated-population share: The ceiling is global CF patient counts and the proportion with ongoing airway mucus burden despite CFTR modulation.
• Persistence of DNase alfa use in treated CF populations: Real-world prescribing depends on phenotype, exacerbation history, lung function trajectory, and clinician/payer acceptance.
• Geography and payer coverage: Uptake and continuity track reimbursement rules, biosimilar/therapeutic alternatives, and local formulary status.

Market constraints

• CFTR modulator substitution: In many markets, early initiation of CFTR modulators reduces reliance on supportive inhaled therapies for some patient subgroups.
• Therapy intensity and adherence: Daily inhalation therapy creates adherence friction, influencing real-world persistence.

Practical market segmentation

For projection modeling, the market breaks into:

• CF patients using chronic supportive airway therapies (including DNase alfa)
• CF patients with inadequate response or residual lung disease on top of CFTR modulators
• Geographic and payer segments where supportive inhaled therapies remain standard of care

Who competes with PULMOZYME and how does that affect pricing and uptake?

Primary competitive set (therapeutic class overlap)

• Other inhaled mucolytics / airway clearance adjuncts
• CFTR modulators (substitute in some patients)
• Anti-inflammatory respiratory therapies used in CF care pathways

Competitive dynamics that typically move the needle

• Switching behavior: Patients with strong CFTR modulator response may de-escalate supportive therapies.
• Add-on positioning: DNase alfa often remains an “add-on” where mucus burden persists.
• Formulary decisions: Payers can pressure step therapy or restrict use to specific clinical criteria (exacerbation frequency, FEV1 thresholds, or documented benefit).

Market analysis: what the commercial pattern usually looks like for older CF respiratory biologics

Even without a fresh trial census, PULMOZYME’s commercial pattern follows a well-observed trajectory in CF:

1. Peak adoption period when DNase alfa is widely used in standard CF supportive care.
2. Modulator era compression where utilization falls for some cohorts.
3. Residual growth pockets driven by phenotype, inadequate CFTR response, and ongoing mucus-driven morbidity.

Implications for investors and planners

• Unit sales tend to be relatively stable in geographies with strong chronic-care coverage but face downward pressure as modulator coverage broadens.
• Net price realization depends on reimbursement and tender/contract dynamics; older biologics often experience price normalization.
• Major upside comes from maintaining guideline-aligned use in residual disease cohorts rather than broad expansion.

How should you project future revenue and demand (framework for PULMOZYME)?

A projection must be quantitative to be decision-grade. Without verified inputs (current units, global net sales by geography, payer restrictions, patient counts, and current utilization rates), a numeric forecast risks being wrong. No numeric projection is provided here.

Non-numeric projection logic (decision-grade)

Forecast directionally from four levers:

• Patient cohort size: CF prevalence and survival trends change the treated pool.
• Modulator penetration: Higher uptake reduces DNase alfa share unless it remains indicated for residual disease.
• Persistence (treatment continuation rate): Adherence and clinician switching drive retention.
• Formulary and pricing: Contracting and reimbursement determine net revenue per patient.

Evidence-based utilization criteria commonly used in CF supportive therapy (how it impacts PULMOZYME demand)

In reimbursement practice, DNase alfa coverage often depends on documented CF diagnosis and clinical justification tied to pulmonary status. This creates a “coverage gating” effect that can sustain use even when overall CF supportive therapy intensity declines.

Key takeaways

• PULMOZYME (dornase alfa) is a legacy CF inhaled therapy whose market demand tracks residual mucus-driven disease burden rather than CF care “baseline” alone.
• The clinical trial update cannot be completed here to a registry-accurate standard without trial-status inputs.
• Market projection requires validated current sales, treated-patient counts, geography, and utilization rates; without those, only directional logic is actionable.
• In the modulator era, substitution pressure is real, but add-on use in residual disease can preserve a durable demand base.

---

FAQs

1) What is PULMOZYME used for?

It is used in cystic fibrosis to improve lung function and support airway mucus clearance.

2) Does CFTR modulator use reduce PULMOZYME demand?

Often, it reduces DNase alfa use in some patient subgroups, while it can sustain use for patients with residual lung disease who continue to need supportive airway therapy.

3) What is the main competitive threat to PULMOZYME?

The largest substitution risk comes from CFTR modulators, with additional pressure from overlapping airway clearance and respiratory supportive therapies.

4) What determines whether patients stay on PULMOZYME?

Clinician assessment of ongoing mucus burden, lung function trajectory, exacerbation history, adherence, and payer coverage criteria.

5) What is the biggest driver for revenue changes going forward?

Net utilization in the “residual disease” cohort and the speed and breadth of CFTR modulator penetration across markets.

---

References

[1] PULMOZYME (dornase alfa) product information and prescribing information (FDA label).