Pulmozyme for CF

Last updated Jan. 18, 2023, by Teresa Carvalho, MS

✅ Fact-checked by José Lopes, PhD


What is Pulmozyme for CF

Pulmozyme (dornase alfa) is an approved oral inhalation solution that acts as a mucolytic, or mucus-thinning agent, to improve lung function in people with cystic fibrosis (CF).

The treatment, marketed by Genentech (part of the Roche group), is indicated for use alongside other CF medications.

Tigerase is a biosimilar of Pulmozyme, which means the treatment, by Generium, has the same therapeutic activity as the original. The biosimilar became available in Russia in 2019.

How does Pulmozyme work?

CF is caused by mutations in a gene called CFTR. This gene codes for a protein that works as a channel in the transport of salts and water in and out of cells. Mutations in CFTR cause the protein to be made incorrectly, or not at all; the resulting lack of the proper protein causes thick, sticky mucus to build up in different tissues and organs, interfering with their function. In the lungs, that buildup makes it difficult for patients to breathe, as well as making them more susceptible to infections.

The mucus in CF patients’ lungs contains long strands of DNA that have been left behind by cells. Pulmozyme contains an enzyme called a DNase that cuts the long DNA strands into smaller pieces. This makes the mucus thinner and easier to cough up. Thinning the mucus also may make CF patients less susceptible to infections.

Who can take Pulmozyme?

Pulmozyme was initially approved by the U.S. Food and Drug Administration (FDA) in 1993. It was the first drug developed specifically for CF.

The therapy can be used by children and adults with CF and is available in more than 65 countries, including Brazil, South Africa, Canada Australia, and New Zealand.

Who should not take Pulmozyme?

Pulmozyme should not be used in patients with known hypersensitivity to:

  • its main ingredient, dornase alfa
  • Chinese hamster ovary cell products
  • other components of the therapy.

How is Pulmozyme administered?

Pulmozyme is taken via oral inhalation of an aerosol mist produced by a nebulizer. The medication is available in 2.5 mg/2.5 mL clear, colorless solution contained in single-dose ampules.

The recommended dosage is one 2.5 mg single-use ampule taken once daily. Some patients may benefit from a twice-daily regimen.

Ampules must be squeezed before use to check for possible leaks. After opening the ampule, the patient must use all its contents or discard it. Pulmozyme should not be diluted or mixed with other medications.

The treatment is administered through:

Several jet nebulizers can be used, including:

  • Hudson T Up-draft II and Marquest Acorn II, to be used with the Pulmo-Aide compressor
  • Pari LC Plus or Pari Baby nebulizer (for those who are unable to inhale or exhale orally), to be used with the Pari Proneb compressor
  • Durable Sidestream nebulizer, to be used with a Mobilaire or a PortaNEB compressor.

When using the eRapid nebulizer system, patients are recommended to replace the handset after 90 administrations. This device should only be used by adults or children who are able to use a mouthpiece.

Pulmozyme in clinical trials for CF

Pulmozyme has been studied in two randomized trials in clinically stable CF patients with forced vital capacity (FVC) that’s lower, higher than or equal to 40% of predicted. FVC is a measure of the amount of air that is forcibly exhaled after taking the deepest breath possible.

Trial in patients with FVC higher or equal to 40% of predicted

This study included a total of 968 patients, of whom 325 patients took a placebo, 322 patients received 2.5 mg of Pulmozyme once daily, and 321 participants were given 2.5 mg of the treatment twice a day over 24 weeks, or about six months.

Results showed that Pulmozyme lowered the risk of lung infection, with treated patients having a 27% to 29% reduction in this risk (once daily and twice daily dose, respectively), compared with those on the placebo.

Pulmozyme also significantly improved lung function, with patients showing a 5.6% to 5.8% increase in mean forced expiratory volume in one second (FEV1, the amount of air forcibly exhaled in one second). That increase was seen over six months, compared with the start of treatment with the twice-daily regimen.

Trial in patients with FVC lower than 40% of predicted

This trial involved 158 clinically stable patients treated with a Pulmozyme, at a daily dose of 2.5 mg for 12 weeks, or about three months, and a placebo group comprising 162 patients.

Compared with the placebo, participants treated with Pulmozyme showed a significant improvement in lung function, with a mean change in FEV1 (9.4% vs. 2.1%) and in FVC (12.4% vs. 7.3%). However, Pulmozyme treatment did not lower the risk of having a respiratory tract infection requiring parenteral antibiotics — those given by routes other than the digestive tract.

Trials in children

The safety and efficacy of Pulmozyme in patients ages 5 to 17 was assessed in a randomized, placebo-controlled trial of 303 CF children within that age range.

In children younger than 5, treatment approval was supported by extrapolating the efficacy results of older children. That group involved children ages 5 and older, with further safety data collected from 65 children ages 3 months to 4 years who were treated with Pulmozyme at a daily dose of 2.5 mg for two weeks.

The safety of Pulmozyme at 2.5 mg, given daily by inhalation over two weeks, was studied in 98 CF children ages 3 months to 10 years. Overall, the nature of the side effects was similar to that seen in the placebo-controlled trials in older patients. More patients reported cough, rash and rhinits — meaning nasal congestion and runny nose — in the younger age group than in the older group.

Comon side effects of Pulmozyme

The most common side effects associated with Pulmozyme are:

  • voice alteration
  • sore throat
  • skin rash
  • chest pain
  • pink eye
  • rhinitis
  • fever
  • shortness of breath.

Use in pregnancy and breastfeeding

Based on animal data, there is no evidence of fetal harm at doses approximately 600 times the maximum recommended human dose.

However, since there are no adequate and well-controlled studies in pregnant human patients, individuals planning to become pregnant or breastfeed should discuss this issue with their healthcare team.

 


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