Efficacy and Safety Data for Pulmozyme

Pulmozyme is proven to reduce the risk of exacerbation and improve lung function with once or twice-daily dosing.


Exacerbation Rate Data

Clinically proven reduction of exacerbation risk in patients with an FVC ≥40% of predicted.1,2 Patients with early CF disease (FVC >85% of predicted) also benefited from Pulmozyme.1

Once-daily (QD) dosing: Pulmozyme clinical efficacy data1

Pulmozyme QD reduced the risk of CF exacerbations among patients ≥5 years of age with FVC ≥40% of predicted—including those with early CF disease (FVC >85% of predicted).1

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27%

risk reduction for exacerbations with QD dosing (P=0.015)1

Proportion of patients remaining exacerbation-free over 6 months10

Proportion of patient remaining exacerbation-free over 6 months

*An exacerbation is defined as a respiratory tract infection requiring parenteral antibiotics.

Twice-daily (BID) dosing: Pulmozyme clinical efficacy data1

When once-daily dosing isn't enough, keep appropriate patients fighting with Pulmozyme BID. Pulmozyme BID reduced the relative risk of exacerbations among patients ≥5 years of age with FVC ≥40% of predicted

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29%

risk reduction for exacerbations with BID dosing1 
Relative risk vs placebo, 0.71; P=0.007

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In patients with FVC ≥40% of predicted, Pulmozyme works to reduce risk of exacerbation even if patients don’t feel its effects1

If your patients over 21 years of age on Pulmozyme QD are experiencing exacerbations, consider BID dosing to help reduce their risk of further exacerbations1

IMPORTANT SAFETY INFORMATION

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

Incidence of first exacerbation1

  All patients (N=968) Baseline FVC >85% (N=370)
Placebo (n) 43% (325) 27% (131)
2.5 mg QD (n) 34% (322) 21% (121)
2.5 mg BID (n) 33% (321) 14% (118)

Adverse Reactions that Occurred in a Higher Proportion (≥3%) of Pulmozyme-Treated Patients than Placebo1

Trial 1: CF Patients with FVC ≥40% of predicted treated for 24 weeks

Adverse Reactions (of any severity or seriousness) Placebo n=325 Pulmozyme QD n=322 Pulmozyme BID n=321
Voice alteration 7% 12% 16%
Pharyngitis 33% 36% 40%
Rash 7% 10% 12%
Laryngitis 1% 3% 4%
Chest pain 16% 18% 21%
Conjunctivitis 2% 4% 5%

Trial 1: CF Patients with FVC ≥40% of predicted treated for 24 weeks

Adverse Reactions (of any severity or seriousness) showing differences less than 3% for:
  • Rhinitis
  • FVC decrease of ≥ 10% of predicted
  • Fever
  • Dyspepsia
  • Dyspena (when reported as serious)

Lung Function Data

Whether your patients with CF need once-daily or twice-daily dosing, fight CF with clinically proven lung function improvement

Once-daily (QD) dosing: Pulmozyme clinical trial data

Pulmozyme QD provided rapid and sustained lung function improvement.1,9

FEV1 change from baseline over 6 months in patients with FVC ≥40% of predicted9,10

5.8% FEV improvement at 6 month treatment duration
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Within 8 days of the start of treatment, mean FEV1 levels increased by 7.9% from baseline1

Twice-daily (BID) dosing: Pulmozyme clinical trial data

When once-daily dosing isn't enough, keep appropriate patients fighting with Pulmozyme BID. Pulmozyme BID provided rapid and sustained lung function improvement.

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Over 6 months: Mean FEV1 levels increased by 5.6% from baseline1,9

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Within 8 days of the start of treatment: Mean FEV1 levels increased by 9% from baseline1

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Pulmozyme works to help improve lung function even if patients don’t feel its effects1

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.

Important Safety Information

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 have been established in pediatric patients 5 years of age and older. The safety of Pulmozyme, 2.5 mg by inhalation, was studied with 2 weeks of daily administration in 65 patients with cystic fibrosis aged 3 months to < 5 years. While clinical trial data are limited in pediatric patients younger than 5 years of age, the use of Pulmozyme should be considered for pediatric CF patients who may experience potential benefit in pulmonary function or who may be at risk of respiratory tract infection.

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 BABY™ 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 full Pulmozyme Prescribing Information for additional Important Safety Information.

    • Pulmozyme [package insert]. South San Francisco, CA: Genentech, Inc; 2018.

      Pulmozyme [package insert]. South San Francisco, CA: Genentech, Inc; 2018.

    • 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/Our-Research/Our-Research-Approach/Research-Milestones. August 17, 2020.

      Research milestones. Cystic Fibrosis Foundation website. https://www.cff.org/Our-Research/Our-Research-Approach/Research-Milestones. 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; 2019. 

      Trikafta [Package insert]. Boston, MA: Vertex Pharmaceuticals Incorporated; 2019. 

    • 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; 2018.

      Pulmozyme Instructions for Use. South San Francisco, CA: Genentech, Inc; 2018.

    Non-US Residents visit: Pulmozyme.global