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Multitherapy approach
Pulmozyme MOA

Each therapy plays a unique role in fighting CF, but only Pulmozyme targets extracellular DNA to break up and thin mucus11,19

CFTR (cystic fibrosis transmembrane conductance regulator) modulators and Pulmozyme help fight CF in different ways. CFTR modulators and Pulmozyme both work to reduce the amount of thick, sticky mucus in the lungs and each is an important part of the multitherapy approach to managing CF. 

CFTR Modulators before and after
Before & After

CFTR Modulators20

Oral medications that help chloride ion conduction by modulating the defective CFTR channel protein in the cells lining the lungs and other organs.

Pulmozyme® (dornase alfa) before and after
Before & After

Pulmozyme1,6,11

Pulmozyme is inhaled and goes directly to the lungs to help reduce viscosity by cleaving extracellular DNA. This breaks up sticky mucus, making it looser and thinner.

In phase 3 clinical trials of Trikafta©,  all patients agreed to remain on their standard-of-care treatment, including Pulmozyme15-17

In the clinical studies that supported the recent FDA approval of Trikafta© (elexacaftor/tezacaftor/ivacaftor and ivacaftor), patients agreed to remain on their standard-of-care CF therapies such as Pulmozyme and other treatments.

93% of patients

In a clinical trial, 93% (99/107) of patients had Pulmozyme as part of their treatment regimen16

81% of patients

In another clinical trial, 81% (326/403) of patients had Pulmozyme as part of their treatment regimen17

* These trials were not designed to assess the efficacy and safety of these agents in combination with Pulmozyme.15
Trikafta is a registered trademark of Vertex Pharmaceuticals Inc.

All CF therapies target different disease mechanisms

Pulmozyme® before and after
Before & After

Pulmozyme1,11

Can help reduce mucus viscosity by cleaving extracellular DNA, and breaking up sticky mucus

CFTR Modulators before and after
Before & After

CFTR Modulators20

Improve chloride ion conduction by modulating the defective CFTR channel protein

Hydrators before and after
Before & After

Hydrators7

Restore airway surface liquid that has been depleted

Airway Clearance Techniques before and after
Before & After

Airway Clearance Techniques21

Move mucus through the airways to help counter defective mucociliary clearance

Antibiotics before and after
Before & After

Antibiotics22

Kill bacteria and help prevent and treat infection

Bronchodilators before and after
Before & After

Bronchodilators22

Help open airways more by relaxing the airway lining, which has become inflamed due to structural damage/bronchiectasis

The only FDA-approved mucus-altering therapy, Pulmozyme plays a distinct role in the fight against cystic fibrosis1,7,14

DNA-laden mucus leads to a cycle of obstruction, infection, and inflammation. The buildup of material called extracellular DNA makes the mucus in the lungs even thicker than before, making it more difficult to clear the lungs properly. As mucus continues to get thicker, more bacteria become trapped, which can eventually lead to a respiratory tract infection. As white blood cells attempt to fight the germs caught in the mucus, they leave behind extracellular DNA. The buildup of these remains leads to further obstruction and lung damage.2-5

How Pulmozyme works to fight CF

 

  • Pulmozyme breaks up mucus and affects the CF cycle of obstruction, infection, and inflammation2-5
  • This action has been shown to reduce sputum viscoelasticity, allowing patients with CF to clear mucus more easily2-5

 

Pulmozyme is the only FDA-approved mucolytic agent that breaks up and thins mucus7

The distinct mechanism of action (MOA) of Pulmozyme breaks up mucus and affects the CF cycle of obstruction, infection, and inflammation

Pulmozyme® (dornase alfa) Mechanism of Action (MoA) Image: Cross section of lung airway.

In CF, thick mucus laden with extracellular DNA hinders mucociliary clearance, contributing to the cycle of obstruction, infection, and inflammation that ultimately leads to reduced lung function.

Pulmozyme® (dornase alfa) Mechanism of Action (MoA) Image: Pulmozyme is the only FDA-approved mucolytic that breaks up mucus.

Pulmozyme is the only FDA-approved mucolytic that breaks up mucus to affect the obstruction-infection-inflammation cycle.

Pulmozyme® (dornase alfa) Mechanism of Action (MoA) Image: In vitro, Pulmozyme cleaves neutrophil-derived DNA.

In vitro, Pulmozyme cleaves neutrophil-derived DNA.1-5

Pulmozyme® (dornase alfa) Mechanism of Action (MoA) Image: This action has been shown to reduce sputum viscoelasticity, allowing cystic fibrosis (CF) patients to clear mucus more easily.

This action has been shown to reduce sputum viscoelasticity, allowing patients with CF to clear mucus more easily.

Pulmozyme targets extracellular DNA to break up and loosen CF mucus1,7

Cystic Fibrosis (CF) mucus before Pulmozyme® (dornase alfa)

CF mucus before Pulmozyme

Cystic Fibrosis (CF) mucus after Pulmozyme® (dornase alfa)

CF mucus after Pulmozyme

Pulmozyme is a mucolytic that, in preclinical laboratory studies, was shown to target and cleave extracellular DNA to decrease mucus viscosity. It is a solution of recombinant human deoxyribonuclease I, an enzyme with a primary amino acid sequence that is identical to the native human enzyme.

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Next Page: Clinical Results

Important Safety Information

Indication and Usage

Pulmozyme (dornase alfa) is indicated for daily administration in conjunction with standard therapies for the management of cystic fibrosis (CF) patients to improve pulmonary function.

In CF patients with an FVC ≥ 40% of predicted, daily administration of Pulmozyme has also been shown to reduce the risk of respiratory tract infections requiring parenteral antibiotics.

Pulmozyme is contraindicated in patients with known hypersensitivity to dornase alfa, Chinese Hamster Ovary cell products, or any component of the product.

The most common adverse reactions associated with the use of Pulmozyme include: voice alteration, pharyngitis, rash, laryngitis, chest pain, conjunctivitis, rhinitis, decrease in FVC of ≥ 10%, fever, dyspepsia, and dyspnea. There have been no reports of anaphylaxis attributed to the administration of Pulmozyme. Mild to moderate urticaria and mild skin rash have been observed and have been transient.

Pediatric Use

The safety and effectiveness of Pulmozyme in conjunction with standard therapies for cystic fibrosis have been established in pediatric patients. Use of Pulmozyme in pediatric patients is supported by evidence in the following age groups:

  • Patients 5 to 17 years of age: A randomized, placebo-controlled trial of 303 of clinically stable cystic fibrosis patients 5 to 17 years of age who received Pulmozyme.
  • Patients less than 5 years: Extrapolation of efficacy data in patients 5 years of age and older with additional safety data in 65 pediatric patients aged 3 months to less than 5 years who received Pulmozyme 2.5 mg daily by inhalation for 2 weeks.

The safety of Pulmozyme, 2.5 mg by inhalation, was studied with 2 weeks of daily administration in 98 pediatric patients with cystic fibrosis 3 months to 10 years of age (65 aged 3 months to < 5 years, 33 aged 5 to ≤ 10 years). The PARI BABYTM reusable nebulizer (which uses a facemask instead of a mouthpiece) was utilized in patients unable to demonstrate the ability to inhale or exhale orally throughout the entire treatment period (54/65, 83% of the younger; and 2/33, 6% of the older patients). Overall, the nature of adverse reactions was similar to that seen in the placebo-controlled trials in older patients. The number of patients reporting cough was higher in the younger age group as compared to the older age group (29/65, 45%; compared to 10/33, 30%) as was the number reporting moderate to severe cough (24/65, 37%; compared to 6/33, 18%). The number of patients reporting rhinitis was higher in the younger age group as compared to the older age group (23/65, 35%; compared to 9/33, 27%) as was the number reporting rash (4/65, 6% as compared to 0/33, 0%).

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

Please see Pulmozyme full Prescribing Information for additional Important Safety Information.

    • Pulmozyme (dornase alfa) [package insert]. South San Francisco, CA: Genentech, Inc; 2024.

      Pulmozyme (dornase alfa) [package insert]. South San Francisco, CA: Genentech, Inc; 2024.

    • Harms HK, Matouk E, Tournier G, et al; DNase International Study Group. Multicenter, open-label study of recombinant human DNase in cystic fibrosis patients with moderate lung disease. Pediatr Pulmonol. 1998;26(3):155-161.

      Harms HK, Matouk E, Tournier G, et al; DNase International Study Group. Multicenter, open-label study of recombinant human DNase in cystic fibrosis patients with moderate lung disease. Pediatr Pulmonol. 1998;26(3):155-161.

    • Chmiel JF, Konstan MW. Anti-inflammatory medications for cystic fibrosis lung disease: selecting the most appropriate agent. Treat Respir Med. 2005;4(4):255-273.

      Chmiel JF, Konstan MW. Anti-inflammatory medications for cystic fibrosis lung disease: selecting the most appropriate agent. Treat Respir Med. 2005;4(4):255-273.

    • Puchelle E, de Bentzmann S, Zahm JM. Physical and functional properties of airway secretions in cystic fibrosis—therapeutic approaches. Respiration. 1995;62(suppl 1):2-12.

      Puchelle E, de Bentzmann S, Zahm JM. Physical and functional properties of airway secretions in cystic fibrosis—therapeutic approaches. Respiration. 1995;62(suppl 1):2-12.

    • King M. Mucolytics and mucus clearance. In: Rubin BK, van der Schans CP, eds. Therapy for Mucus-Clearance Disorders. New York, NY: Marcel Dekker Inc; 2004:201-224.

      King M. Mucolytics and mucus clearance. In: Rubin BK, van der Schans CP, eds. Therapy for Mucus-Clearance Disorders. New York, NY: Marcel Dekker Inc; 2004:201-224.

    • Flume PA, Van Devanter DR. State of progress in treating cystic fibrosis respiratory disease. BMC Med. 2012;10:88.

      Flume PA, Van Devanter DR. State of progress in treating cystic fibrosis respiratory disease. BMC Med. 2012;10:88.

    • Drug development pipeline. Cystic Fibrosis Foundation website. https://www.cff.org/trials/pipeline. Accessed August 17, 2020.

      Drug development pipeline. Cystic Fibrosis Foundation website. https://www.cff.org/trials/pipeline. Accessed August 17, 2020.

    • Research milestones. Cystic Fibrosis Foundation website. https://www.cff.org/about-us/our-history. August 17, 2020.

      Research milestones. Cystic Fibrosis Foundation website. https://www.cff.org/about-us/our-history. August 17, 2020.

    • Fuchs HJ, Borowitz DS, Christiansen DH, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med. 1994;331(10):637-642.

      Fuchs HJ, Borowitz DS, Christiansen DH, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. N Engl J Med. 1994;331(10):637-642.

    • Data on file. Genentech, a member of the Roche Group.

      Data on file. Genentech, a member of the Roche Group.

    • Cystic Fibrosis Foundation Patient Registry 2016 Annual Data Report. Bethesda, MD. ©2017 Cystic Fibrosis Foundation.

      Cystic Fibrosis Foundation Patient Registry 2016 Annual Data Report. Bethesda, MD. ©2017 Cystic Fibrosis Foundation.

    • Sly PD, Brennan S, Gangell C, et al. Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. Am J Respir Crit Care Med. 2009;180(2):146-152. 

      Sly PD, Brennan S, Gangell C, et al. Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. Am J Respir Crit Care Med. 2009;180(2):146-152. 

    • Sly PD, Gangell CL, Chen L, et al. Risk factors for bronchiectasis in children with cystic fibrosis. N Engl J Med. 2013;368(21):1963-1970.

      Sly PD, Gangell CL, Chen L, et al. Risk factors for bronchiectasis in children with cystic fibrosis. N Engl J Med. 2013;368(21):1963-1970.

    • Bakker EM, Volpi S, Salonini E, et al. Small airway deposition of dornase alfa during exacerbations in cystic fibrosis; a randomized controlled clinical trial. Pediatr Pulmonol. 2020;49(2):154-161.

      Bakker EM, Volpi S, Salonini E, et al. Small airway deposition of dornase alfa during exacerbations in cystic fibrosis; a randomized controlled clinical trial. Pediatr Pulmonol. 2020;49(2):154-161.

    • Trikafta [Package insert]. Boston, MA: Vertex Pharmaceuticals Incorporated; 2021. 

      Trikafta [Package insert]. Boston, MA: Vertex Pharmaceuticals Incorporated; 2021. 

    • Heijerman HGM, McKone EF, Downey DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019;394(10212)1940-1948. 

      Heijerman HGM, McKone EF, Downey DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019;394(10212)1940-1948. 

    • Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809-1819. 

      Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809-1819. 

    • Pulmozyme Instructions for Use. South San Francisco, CA: Genentech, Inc; 2024.

      Pulmozyme Instructions for Use. South San Francisco, CA: Genentech, Inc; 2024.

    • Mogayzel PJ, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187:680-689.

      Mogayzel PJ, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187:680-689.

    • Clancy JP. Ongoing research into CFTR modulation. Adv Stud Med. 2010;10(1):14-18.

      Clancy JP. Ongoing research into CFTR modulation. Adv Stud Med. 2010;10(1):14-18.

    • Treatments and therapies. Cystic Fibrosis Foundation website. https://www.cff.org/Life-With-CF/Treatments-and-Therapies/. Accessed July 22, 2021. 

      Treatments and therapies. Cystic Fibrosis Foundation website. https://www.cff.org/Life-With-CF/Treatments-and-Therapies/. Accessed July 22, 2021. 

    • Medications. Cystic Fibrosis Foundation website. https://www.cff.org/Life-With-CF/Treatments-and-Therapies/Medications/. Accessed July 22, 2021.

      Medications. Cystic Fibrosis Foundation website. https://www.cff.org/Life-With-CF/Treatments-and-Therapies/Medications/. Accessed July 22, 2021.

    Non-US Residents visit: Pulmozyme.global