Wednesday, Dec 2, 1992
South San Francisco, Calif. -- December 2, 1992 --Genentech, Inc. (NYSE: GNE) announced today the completion of its Phase III clinical trial of Pulmozyme (rhDNase) in the treatment of cystic fibrosis. The results of the 968 patient study demonstrated Pulmozyme improves lung function and reduces the rate of respiratory infection in patients with cystic fibrosis. No major adverse side effects were noted.
Genentech is proceeding quickly to complete and file a Product License Application (PLA) with the Center for Biologics Evaluation and Research (CBER) of the Food and Drug Administration (FDA), and is also planning a simultaneous submission to the Bureau of Biologics, Health Protection Branch in Canada. The filing of the PLA is expected to be completed by March 1993. Similar filings with relevant regulatory agencies in Europe are expected shortly thereafter.
A thorough analysis of the Phase III study will be presented at the Intermountain Thoracic Society Meeting to be held in Snowbird, Utah on January 26, 1993. The study is also being developed for submission to a peer-reviewed medical journal for publication.
Cystic fibrosis is the most common fatal genetic disease in the United States. Individuals with cystic fibrosis have thick lung secretions that cause impaired breathing, frequent lung infections, and continual degeneration of lung function. Median survival age for cystic fibrosis patients is 29 years. There are an estimated 30,000 Americans, almost 3,000 Canadians, and 20,000 Europeans with cystic fibrosis.
According to Robert K. Dresing, President of the Cystic Fibrosis Foundation, "These results are promising for patients and their families, but it is vital to understand that this product is not commercially available yet, and this clinical data must proceed through what we hope will be an expeditious but very important scientific and regulatory review process."
At Genentech's headquarters in South San Francisco, personnel are working extended shifts to complete construction of a state-of-the-art manufacturing facility to produce commercial quantities of Pulmozyme. Start-up of the facility is planned to begin in first quarter 1993. It will then require inspection and licensing approval by regulatory agencies. Until licensing is complete, product availability will remain very limited. "We do not anticipate any delay in commercialization of Pulmozyme due to manufacturing considerations," emphasized G. Kirk Raab, President and CEO of Genentech.Prior to commercial approval of Pulmozyme, Genentech is committed to working with regulatory authorities and the cystic fibrosis medical community to provide an expanded access program for the most appropriate patients using the limited amount of product that is available. "Expanded access" is a term used to describe availability of a product to physicians prior to commercial availability for selected patients who have not been in previous clinical trials.
"But the clinical selection criteria and very limited supply of product will make that program available to only a small number of patients," explained Raab. "We ask for the continued patience and understanding of patients and their families as we do everything we can to get the new manufacturing facility on-line and expedite the submission of the PLA."
Details of the expanded access program will be announced when the results of an ongoing study due to be completed in early 1993 in seriously ill cystic fibrosis patients have been analyzed, and when issues of product availability and selection criteria have been worked out with the advice of the FDA and outside medical experts. Actual implementation of an expanded access program is not anticipated until first quarter 1993.Genentech, Inc. is a leading biotechnology company that discovers, develops, manufactures and markets human pharmaceuticals for significant 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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