Tuesday, Jan 29, 1991
South San Francisco, Calif. -- January 29, 1991 --Genentech, Inc. announced today that it had received an official notification of designation from the U.S. Food and Drug Administration (FDA) granting orphan drug status for Genentech's recombinant human DNase for the management of cystic fibrosis.
Under the Orphan Drug Act, human pharmaceuticals sold for profit may be granted orphan drug status if they are indicated for patient populations of less than 200,000. More than one company may be granted orphan drug status while the drug is under development, but the first company to obtain approval for a designated drug receives U.S. marketing exclusivity for seven years for the approved indication.
Cystic fibrosis afflicts approximately 30,000 children and young adults who usually die from respiratory failure brought on by repeated lung infections. Persistent, thick secretions which clog the lungs are a frequent occurrence in patients with cystic fibrosis, the most common fatal genetic disease in the United States. Median survival age for cystic fibrosis patients is 28 years. To date, no approved therapy exists that effectively shortens the course of lung infections by dissolving excessive and thick infected mucus secretions.
"Without the fundamental incentives of the Orphan Drug Act, it would have been very difficult for Genentech to develop this promising potential treatment to fight the devastating effects of cystic fibrosis," said G. Kirk Raab, president and chief executive officer of Genentech.
Phase I studies of DNase, which began last summer, have been completed at two centers: the National Heart, Lung and Blood Institute, which is a division of the National Institutes of Health, and the University of Washington, Seattle. The principal investigators are expected to report the Phase I results early this year. Phase II trials are scheduled to begin during the first half of 1991 at several centers across the country. Genentech is the only company conducting clinical trials for DNase.
DNase is a recombinant form of a naturally-occurring enzyme which dissolves the DNA released by the inflammatory cells that accumulate to fight lung infections. The extracellular DNA contributes to the thick, infected mucus secretions of patients with cystic fibrosis, compromising their normal lung function. Genentech is evaluating the potential of DNase to reduce the viscosity of infected mucus to facilitate removal of the secretions to clear airways. It is hoped that this will improve lung function and help improve and prolong the quality of cystic fibrosis patients' lives.
DNase is still in the early stages of clinical trials and its effectiveness must be proven in further, more extensive clinical trials.
Genentech, Inc. is a biotechnology company focusing on the development, manufacture and marketing of pharmaceutical products produced by recombinant DNA technology.
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