Does Pulmozyme (dornase alpha) cause side effects?

Pulmozyme (dornase alpha) is an inhaled synthetic protein used to treat cystic fibrosis, a lung disease which is the most common fatal genetic disease in developed countries. 

The lungs continually secrete fluid into the airways to keep them moist. In cystic fibrosis, the fluid becomes thick because the amount of water it contains is reduced. The thickened fluid is difficult to cough up or spit out. It blocks the airways, making breathing difficult and promoting the growth of bacteria and infection. 

Infection destroys the tissues of the lungs, and it is the slowly progressive destruction of the lungs that is the major cause of disability and death in children with cystic fibrosis. The thick fluid contains high concentrations of deoxyribonucleic acid (DNA). 

Pulmozyme is a genetically engineered form of the human enzyme, deoxyribonuclease or DNAse. Pulmozyme breaks down the DNA and thereby reduces the thickness of the fluids. 

Common side effects of Pulmozyme include

Serious side effects of Pulmozyme include

Drug interaction studies on Pulmozyme have not been conducted. Pulmozyme is compatible with standard cystic fibrosis therapies including

There are no studies of Pulmozyme in women during pregnancy. Physicians must weigh potential risks with the expected benefits of therapy when deciding whether or not to use Pulmozyme in pregnancy. 

It is unknown if Pulmozyme is secreted in breast milk. At most, very little Pulmozyme would be expected in breast milk after long-term aerosol use. However, because many drugs are secreted in breast milk, physicians must still use caution when they consider the use of Pulmozyme in breastfeeding women.

What are the important side effects of Pulmozyme (dornase alpha)?

The most common side effects of dornase alpha include:

Pulmozyme (dornase alpha) side effects list for healthcare professionals

Clinical Trials Experience

  • Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
  • The data described below reflect exposure to Pulmozyme in 902 patients, with exposures ranging from 2 weeks of daily administration up to once or twice daily administration for six months.
  • Pulmozyme was studied in both placebo-controlled and uncontrolled trials (n=804 and n=98).
  • The population of patients in placebo-controlled trials was with FVC ≥ 40% of predicted (n=643) or with more advanced pulmonary disease, FVC < 40% of predicted (n=161).
  • The population in the uncontrolled trial included 98 pediatric patients with CF ranging from 3 months to 10 years of age.
  • More than half of the patients received Pulmozyme 2.5 mg by inhalation once a day (n=581), while the rest of patients (n=321) received Pulmozyme 2.5 mg by inhalation twice a day.
Placebo-Controlled Trials
  • Trial 1: Trial 1 was a randomized, placebo-controlled clinical trial in patients with FVC ≥ 40% of predicted. In this trial, over 600 patients received Pulmozyme once or twice daily for six months.
  • The most common adverse reaction (risk difference ≥5%) was voice alteration. The proportion of most adverse events was similar for patients on Pulmozyme and on placebo, probably reflecting the sequelae of the underlying lung disease.
  • In most cases reactions that were increased were mild, transient in nature, and did not require alterations in dosing.
  • Few patients experienced adverse reactions resulting in permanent discontinuation from Pulmozyme, and the proportion of discontinuations were similar for placebo (2%) and Pulmozyme (3%).
  • Adverse reactions occurring in a higher proportion (greater than 3%) of Pulmozyme treated patients than in placebo-treated patients are listed in Table 2.
  • Trial 2: Trial 2 was a randomized, placebo-controlled trial in patients with more advanced pulmonary disease (FVC < 40% of predicted) who were treated for 12 weeks.
  • In this trial, the safety profile of Pulmozyme was similar to that reported in patients with less advanced pulmonary disease (FVC ≥ 40% of predicted).
  • Adverse reactions that were reported in this trial with a higher proportion (greater than 3%) in the Pulmozyme treated patients are listed in Table 2.

Table 2: Adverse Reactions Increased 3% or More in Pulmozyme Treated Patients Over Placebo in CF Clinical Trials

Adverse Reactions (of any severity or seriousness)Trial 1 CF Patients with FVC ≥ 40% of predicted treated for 24 weeksTrial 2 CF Patients with FVC <40% of predicted treated for 12 weeks
Placebo
n=325
Pulmozyme QD
n=322
Pulmozyme BID
n=321
Placebo
n=159
Pulmozyme QD
n=161
Voice alteration7%12%16%6%18%
Pharyngitis33%36%40%28%32%
Rash7%10%12%1%3%
Laryngitis1%3%4%1%3%
Chest Pain16%18%21%23%25%
Conjunctivitis2%4%5%0%1%
Rhinitis24%30%
FVC decrease of ≥ 10% of predicted*Differences were less than 3%17%22%
Fever28%32%
Dyspepsia0%3%
Dyspnea (when reported as serious)Differences were less than 3%12%†17%†
*Single measurement only, does not reflect overall FVC changes.
† Total reports of dyspnea (regardless of severity or seriousness) had a difference of less than 3% in Trial 2.

  • Mortality rates observed in controlled trials were similar for the placebo and Pulmozyme treated patients.
  • Causes of death were consistent with progression of cystic fibrosis and included
Uncontrolled Trial
  • Trial 3: 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.
  • 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% as 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).
Allergic Reactions
  • There have been no reports of anaphylaxis attributed to the administration of Pulmozyme.
  • Urticaria, mild to moderate, and mild skin rash have been observed and have been transient.
  • Within all of the studies, a small percentage (average of 2-4%) of patients treated with Pulmozyme developed serum antibodies to Pulmozyme.
  • None of these patients developed anaphylaxis, and the clinical significance of serum antibodies to Pulmozyme is unknown.

Postmarketing Experience

  • Postmarketing spontaneous reports and prospectively collected safety data from observational studies confirm the safety profile to be as described in clinical trials.

What drugs interact with Pulmozyme (dornase alpha)?

  • Available data indicate there are no clinically important drug-drug interactions with Pulmozyme.

Treatment & Diagnosis

Medications & Supplements

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Medically Reviewed on 12/9/2020
References
FDA Prescribing Information

Professional side effects and drug interactions sections courtesy of the U.S. Food and Drug Administration.