Monday, Mar 2, 1998
South San Francisco, Calif. -- March 2, 1998 --Genentech Inc. (NYSE: GNE) today announced that a change in labeling for Pulmozyme® (dornase alfa) Inhalation Solution, an aerosol treatment for cystic fibrosis (CF), has been approved by the U.S. Food and Drug Administration (FDA) which includes the safety and alternative administration of Pulmozyme in patients under the age of five. Pulmozyme had been previously approved by the FDA for use in CF patients five years or older.
"I am pleased that the expansion of Pulmozyme's labeling will assist the physician in the earlier treatment of cystic fibrosis, since it is clear that lung disease in CF can begin in the first few years of a patient's life," said Dr. Jeff Wagener, Professor of Pediatrics, University of Colorado, Children's Hospital Denver.
However, because of the limited experience with the administration of Pulmozyme to patients younger than 5 years of age, its use should be considered only for those patients in whom there is a potential for benefit in pulmonary function or for reducing risk of respiratory tract infection.
The safety of 2.5 mg of Pulmozyme® (dornase alfa) Inhalation Solution over two weeks of administration was evaluated in 98 patients with cystic fibrosis ages 3 months to 10 years. Young patients in the trial who were unable to utilize a nebulizer mouthpiece used the PARI BABY -- a reusable nebulizer which is equipped with a tight-fitting facemask.
Adverse events in the under five patient population were similar to those seen in older patients with mild to moderate disease (FVC > 40 percent), including voice alteration, pharyngitis, laryngitis, rash, chest pain and conjunctivitis. However, cough, including moderate to severe cough, rhinitis and rash were reported more frequently in these very young CF patients compared to older children (5 to < 10 years of age).
"Pulmozyme is the first treatment specifically for cystic fibrosis to be shown safe and well tolerated in patients under five years of age," said Arthur D. Levinson, Ph.D., president and chief executive officer of Genentech. "This is significant because it means that Pulmozyme is now available for use in the youngest patients -- infants and toddlers."
CF is an inherited disorder that affects about 25,000 Americans, 3,000 Canadians and 9,000 Europeans. Currently, there are approximately 3,000 patients under five in the United States that have CF. A faulty gene in CF 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. Today, the average life span of patients with CF is 31 years compared to 14 years in 1969.
"This gives us another important tool to intervene early in the disease process," said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation. "It's critical that such a therapy is available for all CF patients."
In December 1993, Pulmozyme® (dornase alfa) Inhalation Solution received approval by the FDA for the management of mild to moderate CF in patients over the age of five in conjunction with standard therapies to reduce the incidence of respiratory tract infections requiring parenteral antibiotics and to improve pulmonary function. In December 1996, the indication for Pulmozyme was expanded to include patients with advanced disease.
Genentech, Inc. is a leading biotechnology company that discovers, develops, manufactures and markets human pharmaceuticals for significant unmet medical needs. Eleven of the currently marketed biotechnology products stem from Genentech science. Genentech markets or promotes eight biotechnology products in the United States. The company has headquarters in South San Francisco, California and is traded on the New York Stock Exchange and Pacific Exchange under the symbol GNE.
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