Pulmozyme Inhalation Solution has been approved for the treatment of cystic fibrosis patients with advanced disease. Pulmozyme originally received marketing clearance by the FDA in December 1993 for the management of mild to moderate cystic fibrosis in conjunction with standard therapies to reduce the incidence of respiratory tract infections requiring parenteral antibiotics and to improve pulmonary function.
Since cystic fibrosis is a disease that worsens over time, this indication supports continued use of Pulmozyme in patients with an FVC of less than 40%.
The benefit of treatment with Pulmozyme in advanced Cystic Fibrosis was demonstrated in a placebo-controlled study of 320 subjects that examined safety and efficacy of Pulmozyme during a 12-week period. Advanced disease is defined by a standard measure of forced vital capacity of less than 40% of predicted (FVC less than 40%). In this study of subjects with advanced disease, measures of pulmonary function--the forced expiratory volume in one second and FVC--were significantly improved as compared to placebo. In contrast to the mild to moderate cystic fibrosis patient population, no benefit reducing the incidence of respiratory tract infections was seen.
Pulmozyme was safe and generally well tolerated in the advanced cystic fibrosis population. Side effects were similar to those seen in patients with mild to moderate disease, including voice alteration, pharyngitis, laryngitis, rash, chest pain, and conjunctivitis. However, rhinitis, fever, a decrease in FVC, dyspepsia, and serious dyspnea were reported more frequently than in the placebo group.
The results of the clinical study were published in the October issue of CHEST.
Cystic Fibrosis is an inherited disorder that affects about 25,000 Americans, 3,000 Canadians, and 20,000 Europeans. A faulty gene in cystic fibrosis patients leads to the production of thick viscous secretions that can cause persistent bacterial infection and congestion. As white blood cells attempt to destroy bacteria, they release DNA, which further thickens the secretions. These thiTk secretions also encourage and prolong respiratory tract infections that damage lung tissue and ultimately lead to death. Today the average life span for patients with cystic fibrosis is 30 years compared to 14 years in 1969.