Wednesday, Oct 9, 1996
South San Francisco, Calif. -- October 9, 1996 --Genentech, Inc. (NYSE: GNE) today announced that Pulmozyme® (dornase alfa) Inhalation Solution, the company's aerosol treatment for cystic fibrosis (CF), was recommended for marketing clearance by the Pulmonary Allergy Drug Advisory Committee to the U.S. Food and Drug Administration (FDA) for treatment of CF patients with advanced disease. It was further recommended that Genentech continue to study long-term outcome of CF patients.
Pulmozyme is currently indicated 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.
The recommendation is based on results of a placebo-controlled study of 320 patients that examined safety and efficacy of Pulmozyme during a twelve-week period in cystic fibrosis patients with advanced lung disease defined by the standard measure of forced vital capacity less than 40% of predicted (FVC 40%). It demonstrated that patients with advanced disease improved significantly as compared to placebo in commonly used measures of pulmonary function -- the forced expiratory volume in one second (FEV1) and FVC. In contrast to the mild to moderate CF patient population, no statistical benefit on respiratory tract infections versus placebo was seen. The results of this study were published in the October issue of CHEST.
Pulmozyme was safe and well tolerated in the advanced CF population. Side effects were similar to those with mild to moderate disease, including voice alteration, pharyngitis, laryngitis, rash, chest pain and conjunctivitis. In addition, rhinitis, a decrease in FVC and dyspepsia were reported more frequently than in the placebo group.
In the United States today, there are approximately 500 cystic fibrosis patients with advanced disease. As CF is a progressive disease, today's recommendation would support continued Pulmozyme therapy of patients once their FVC is less than 40 percent. Many of these patients are still in their teens and life expectancy is two years for half of them.
"We are very pleased with the committee's recommendation, as treatment options for this group of patients are extremely limited, " said Dr. Charles Johnson, clinical scientist, Genentech, Inc. "If cleared by the FDA, the use of Pulmozyme in the treatment of patients with advanced disease fills an unmet medical need while researchers continue their search for a cure," added Dr. Johnson.
CF 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 thick secretions also encourage and prolong respiratory tract infections that damage lung tissue and ultimately lead to death. The median survival age is 30 years.
Genentech, Inc. is a leading biotechnology company that discovers, develops, manufactures and markets human pharmaceuticals for significant unmet medical needs. The company has headquarters in South San Francisco, California, and is traded on the New York and Pacific Stock Exchanges under the symbol GNE.
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