What is Cystic Fibrosis and Pulmozyme® (dornase alfa)
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. This thick mucus can build up in the airways, allowing germs to thrive and become a source of infection and inflammation that can lead to decreased lung function.
As the white blood cells fight the infection caught in the mucus, they leave behind remains called extracellular DNA. The buildup of these remains makes the mucus in lungs affected by CF even thicker than before, leaving your lungs even more vulnerable to decreased lung function. Because the mucus is even more thick and sticky, it can be difficult to get rid of this abnormal mucus.
This may begin a cycle of blockage and infection that may lead to scarring and poorer lung function.
Though you or your loved one may not see or feel the signs and symptoms of CF right away, the disease is still present.
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.