Cystic Fibrosis and Pulmozyme® (dornase alfa) FAQs
It is important to take Pulmozyme as your doctor has prescribed. Pulmozyme has a distinct role in the fight against CF. One of the causes of thickened mucus in the lungs is extracellular DNA. As seen in laboratory studies, Pulmozyme uniquely cuts apart extracellular DNA by acting like an enzyme naturally found in the lungs. Cutting up extracellular DNA can help make mucus thinner and looser, which can make it easier to clear your lungs. In a clinical study of patients over 5 years of age with forced vital capacity (FVC) greater than 40% of predicted, Pulmozyme, when taken once-daily or twice-daily, was shown to improve lung function in most patients. Even when you can't feel Pulmozyme working to fight CF, it is.
Every prescribed therapy plays a role to help you fight CF.
Pulmozyme is a mucolytic. In laboratory studies, Pulmozyme was shown to act like a protein already in your body to cut apart the sticky mucus your lungs produce, making it thinner and looser.
Gene-specific treatments, also called Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators, were studied for use in patients with specific genetic mutations. Even though studies for these gene-specific treatments were not designed to look at the efficacy and safety of CFTR modulators in combination with existing treatments, patients in these studies stayed on their prescribed CF therapies (eg, Pulmozyme, tobramycin).
Please contact your doctor and/or cystic fibrosis (CF) Care Team to discuss your specific treatment plan and the distinct role each of your prescribed therapies plays.
Pulmozyme was approved by the US Food and Drug Administration (FDA) in 1993, and was the first drug developed specifically for cystic fibrosis (CF).
Patients may experience the following when using Pulmozyme: change in or loss of voice, throat discomfort, rash, chest pain, red watery eyes, runny nose, lowering of lung function, fever, indigestion, and shortness of breath. There have been no reports of severe allergic reactions caused by the administration of Pulmozyme. Mild to moderate hives and mild skin rash have been observed and have been short-lived.
Pulmozyme is to be used with other standard CF treatments and has demonstrated safety in children over 5 years of age, adolescents, and adults. The efficacy of Pulmozyme has not been studied in patients less than 5 years of age. 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. Overall, the nature of adverse events 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 (45%; compared to 30%) as was the number reporting moderate to severe cough (37%; compared to 18%). The number of patients reporting rhinitis was higher in the younger age group as compared to the older age group (35%; compared to 27%) as was the number reporting rash (6% as compared to 0%).
Pulmozyme should not be used in patients who are allergic to any of its ingredients.
You should call your doctor if you experience any of these side effects or if any of these side effects are severe, get worse, or do not go away.
You may feel more prepared if you talk to your doctor about what to expect before you begin Pulmozyme. Based on studies, patients taking Pulmozyme may experience change in or loss of their voice, discomfort in the throat, rash, chest pain, red watery eyes, runny nose, lowering of lung function, fever, indigestion, and shortness of breath. Mild to moderate hives and mild skin rash have been observed and have been short-lived. You should call your doctor if you experience any of these side effects or if any of these side effects are severe, get worse, or do not go away.
These are not all the possible side effects of Pulmozyme. See the Important Safety Information for more.
Pulmozyme may be an essential partner in your fight against CF. In cystic fibrosis (CF), your lungs produce extra-thick, sticky mucus. Laboratory studies have shown that Pulmozyme makes mucus thinner and looser. Click here to see the clinical data and learn more about whether Pulmozyme is right for you.
The recommended dose for most CF patients is Pulmozyme 2.5 mg once-daily.
Some patients over 21 may benefit from twice-daily treatment. You should ask your doctor whether prescribing Pulmozyme 2.5 mg twice-daily may be right for you.
Treating cystic fibrosis (CF) is complicated so it is important to take all your treatments as prescribed by your doctor. As seen in laboratory studies, Pulmozyme works to help make mucus thinner and looser. Your doctor will prescribe other medicines to take in addition to Pulmozyme. These other medicines may include:
The FDA has approved specific nebulizers/compressors to be used with Pulmozyme.
One nebulizer system is FDA-approved:
The following nebulizers and compressors have been approved for use with Pulmozyme:
Please contact your doctor and/or cystic fibrosis (CF) Care Team for more information about nebulizers and compressors, and for guidance in choosing products that are right for you.
Pulmozyme comes in sterile, single-use ampules that should be used completely. Because Pulmozyme has no preservatives, the entire contents must be used or discarded.
It is important to store ampules of Pulmozyme in their protective foil pouch under refrigeration and protected from light. Refrigerate Pulmozyme during transport, and do not expose it to room temperatures for more than 24 hours.
Each ampule should be squeezed prior to use in order to check for leaks. Discard ampules if the solution is cloudy or discolored. Once opened, the entire contents of the ampule must be used or discarded.
Finding the time and place to take your treatments, like Pulmozyme, isn’t always easy. No one is perfect. But having a routine that fits your lifestyle can help you stay in control and assist in your fight against cystic fibrosis (CF).
There are expiration dates stamped on each ampule. It is important to follow the stamped date and discard any ampules that expire.
Each sterile, single-use ampule of Pulmozyme (2.5 mg) is packaged in a protective foil pouch. The ampules should be left in their foil package and stored in the refrigerator. They should also be kept away from strong light. Refrigerate Pulmozyme during transport, and do not expose it to room temperatures for more than 24 hours.
Cystic fibrosis (CF) is a genetic condition, which means that it is something you are born with. CF is known to cause your lungs to produce extra-thick, sticky mucus. This mucus builds up and clogs your airways.
The lungs naturally produce a thin layer of mucus that helps protect the airways from dangerous bacteria that can cause infections.
For people living with CF, the mucus in the lungs is thicker than normal, and becomes a source of inflammation and infection that can lead to decreased lung function and exacerbation of infections.
Genentech Access Solutions may be able to help you understand how to get the medicine you need.
If you are eligible, there may be options to help you pay for Pulmozyme*:
*Patients must meet certain criteria
Genentech is committed to your treatment as well as your knowledge of cystic fibrosis (CF). A library of resources is available at CF Links.
Pulmozyme (dornase alfa) is indicated for daily administration along 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 injectable antibiotics.
Pulmozyme should not be used in patients who are allergic to any of its ingredients.
Patients may experience the following when using Pulmozyme: change in or loss of their voice, discomfort in the throat, rash, chest pain, red watery eyes, runny nose, lowering of lung function, fever, indigestion, and shortness of breath. There have been no reports of severe allergic reactions caused by the administration of Pulmozyme. Mild to moderate hives and mild skin rash have been observed and have been short-lived.
The safety and effectiveness of Pulmozyme have been established in patients 5 years of age and older. While clinical trial data are limited in patients younger than 5 years of age, the use of Pulmozyme should be considered for pediatric CF patients who may experience potential benefit in lung function or who may be at risk of respiratory tract infection.
The safety of Pulmozyme given by daily inhalation for 2 weeks has been studied using 98 CF patients with 65 of them aged 3 months to <5 years (younger group) and 33 aged 5 years to <10 years (older group). The PARI BABY™ reusable nebulizer (which uses a face mask instead of a mouthpiece) was used in patients who were unable to show that they could breathe in or out using their mouth throughout the entire treatment period. Overall, the kind of side effects observed in children was similar to those seen in larger trials in older patients.
You are encouraged to report side effects to Genentech and the FDA. 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.
For additional Important Safety Information, please click here for the Pulmozyme full Prescribing Information. If you have questions, please discuss them with your CF Care Team.