Thursday, Dec 30, 1993
South San Francisco, Callf. -- December 30, 1993 --Genentech, Inc. announced today that it received a license to market Pulmozyme® (dornase alfa) recombinant human DNase for the treatment of cystic fibrosis (CF) from the Food and Drug Administration (FDA) -- only nine months after the Product License Application was filed.
Pulmozyme is the first new therapeutic approach in 30 years for CF -- the most common fatal genetic disease in Caucasians. Genentech has manufactured ample product supply to meet expected demand and will begin shipping Pulmozyme to distributors in the United States in mid-January, 1994.
Pulmozyme, when used in conjunction with standard therapies, was proven in clinical trials to reduce the risk of respiratory tract infections requiring i.v. antibiotic therapy by 27 percent, maintain an improvement in lung function, reduce costly hospitalizations and other related medical costs and improve quality of life in patients with mild to moderate pulmonary dysfunction.
"Pulmozyme represents a significant advance in the treatment of cystic fibrosis, and it is another example of biotechnology's ability to create a new treatment for a problem that medical researchers have struggled with for decades,. said G. Kirk Raab, Genentech's president and chief executive officer. "The extraordinary pace of Pulmozyme's development shows how effectively industry, patients, caregivers and government regulatory agencies can work together toward medical advances such as this," said Raab.
Pulmozyme® (dornase alfa) is a product of highly sophisticated biotechnology research, development and manufacturing. Its rapid development in five years from test tube to CF patients -- about half the industry average -- is the result of an intense collaboration among Genentech, the Cystic Fibrosis Foundation, CF centers around the world, physicians, patients, the FDA and the Canadian and European regulatory agencies.
Pulmozyme also received a marketing license today in Canada, and has already been approved for marketing in Austria, Sweden and New Zealand. Approval is also expected in the near future in the United Kingdom and shortly thereafter in other major European countries and elsewhere in the world. In Western Europe, Genentech and Roche will work together to commercialize Pulmozyme.
"The approval of Pulmozyme is exciting news for cystic fibrosis patients, because it is going to help make them feel better -- something they dream about every day," said Robert J. Beall, Ph.D., executive vice president for medical affairs, Cystic Fibrosis Foundation. "Finally, CF patients will have a new therapy that will help normalize their lives and offer hope and optimism for the future."
Pulmozyme was shown in clinical trials to improve quality of life for CF patients with mild to moderate pulmonary dysfunction by reducing the need for i.v. antibiotics, hospitalizations and time missed from school, work and other everyday activities. In addition, patients have reported a general improvement in overall wellbeing while on Pulmozyme, saying that they experience less breathlessness, coughing and waking up in the middle of the night to cough or catch their breath.
Due largely to this quality of life improvement, 99 percent of those who completed the Phase III clinical trials chose to remain on or begin (for those taking the placebo) Pulmozyme® (dornase alfa) therapy for the follow-up open label trial.
"Even though Pulmozyme is not a cure or a replacement for current therapies to treat cystic fibrosis, its additive benefits make a significant difference," said one of the drug's principal clinical investigators, Bonnie Ramsey, M.D., Children's Hospital & Medical Center, University of Washington, Seattle. "We are excited to have something to further enhance the outlook and quality of life of our patients."
In the clinical trials, some reported adverse events were more frequent among the 322 patients who received Pulmozyme once-daily than among the 325 placebotreated patients. These were voice alteration (12 percent vs. 7 percent, respectively), sore throat (36 percent vs. 33 percent), laryngitis (3 percent vs. 1 percent), rash (10 percent vs. 7 percent), pain in the chest (18 percent vs. 16 percent) and conjunctivitis (4 percent vs. 2 percent).
Cystic fibrosis, an inherited disorder, affects about 30,000 Americans, 3,000 Canadians and more than 20,000 Europeans. A faulty gene in CF patients leads to the production of thick, viscous secretions, which clog the lungs and make breathing difficult. These thick secretions also tend to cause and prolong respiratory tract infections, which damage lung tissue and ultimately lead to death. The median survival is 29 years of age.
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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