Monday, Aug 9, 1993

FDA Advisory Committee Unanimously Recommends Pulmozyme (DNase) for Approval as Cystic Fibrosis Treatment

South San Francisco, Calif. -- August 9, 1993 --

The Pulmonary Allergy Drugs Advisory Committee of the Food and Drug Administration (FDA) today recommended that the FDA grant marketing approval for Pulmozyme™ (dornase alfa) recombinant human DNase. The independent panel of experts unanimously (7-0) decided in favor of approving Pulmozyme for the treatment of Cystic Fibrosis.

"We are extremely pleased with the Advisory Committee's recommendation to approve Pulmozyme," said G. Kirk Raab, Genentech president and chief executive officer. "This would not have happened without the intense collaboration between Genentech, the FDA, the Cystic Fibrosis Foundation and cystic fibrosis centers and patients across the country," emphasized Raab.

Clinical Trial Results

The Phase III clinical trial -- a randomized, placebo-controlled, doubleblind trial of 968 patients -- showed that Pulmozyme reduces the risk of respiratory tract infections requiring parenteral antibiotic treatment and improves and maintains lung function in patients with cystic fibrosis. The only noteworthy side effects were mild hoarseness, sore throat and rash.

Pulmozyme also offers other significant benefits by reducing the need for antibiotic therapy, decreasing hospital days and time away from work and school due to CF-related illness.

In addition, the Phase 111 clinical trial showed that Pulmozyme enhances patient quality of life by improving breathing, overall well-being (i.e., appetite, activity, sleep, general feeling, energy level), and reducing CF-related symptoms (i.e., cough severity, cough frequency, cough keeping patients awake, chest congestion). The trial also demonstrated that Pulmozyme provides these benefits in the majority of CF patients.

"Today's decision by the FDA Advisory Committee to recommend Pulmozyme for approval is histo.ric in that it brings the CF community significantly closer to having the first new approved therapy for CF patients in nearly three decades," said Robert J. Beall, Ph.D., executive vice president of medical affairs of the Cystic Fibrosis Foundation, during the open public forum of the Advisory Committee Meeting. "Clearly, Pulmozyme will become a major weapon in our fight to control the progressive destruction of this disease."

Genentech has submitted applications for approval in the United States, Canada and Europe. "While the decision made at this meeting is a critical part of the licensing process for Pulmozyme, there are other important elements, such as manufacturing, which need to be approved as well," said M. David MacFarlane, vice president of Regulatory Affairs for Genentech. "Additional time may be needed because of our complex manufacturing process and startup procedure on the new manufacturing facility. We are working closely and productively with the FDA to get Pulmozyme approved and on the market as soon as possible."

Cystic fibrosis, an inherited disorder, is the most common fatal genetic disease in Caucasians, afflicting about 30,000 Americans, nearly 3,000 Canadians, and 20,000 Europeans.

The median survival age is about 29 years. Because of a genetic defect, thick secretions accumulate in the lungs of individuals with cystic fibrosis, causing impaired breathing, persistent lung infection and progressive degeneration of lung function. In these people, DNA is released by white blood cells fighting the infections characteristic of cystic fibrosis, causing the already abnormal secretions to become thicker or more viscous. Sometimes referred to as "molecular scissors," Pulmozyme cuts up the excess DNA in these secretions, helping patients breathe easier and reducing the frequency of respiratory infections.

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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