Genentech Inc. announced Tuesday that it has filed a productlicense application (PLA) with FDA for Pulmozyme, itsrecombinant human DNase, for treating cystic fibrosis.

The South San Francisco, Calif., company also has submittedmarketing applications for Pulmozyme in Europe and Canada.

Genentech (NYSE:GNE) completed its Phase III clinical trials onPulmozyme last December, and reported those results inJanuary at the Intermountain Thoracic Society Meeting.

The 968-patient trial demonstrated that the compound reducesthe rate of respiratory infection (as measured by the need forintravenous antibiotics) and significantly improves lungfunction in cystic fibrosis patients, with no major adverse sideeffects.

Cystic fibrosis (CF) is the most common fatal genetic disease inCaucasians. People with this disease have thick secretions thataccumulate in their lungs, causing impaired breathing,persistent lung infection and progressive degeneration of lungfunction. This chronic damage to the lungs manifests itself as agradual loss in breathing capacity and chronic lung infectionswith periodic hospitalizations.

The trial results indicated that "use of Pulmozyme DNase once aday (2.5 milligrams) resulted in a 31 percent reduction in theuse of parenteral antibiotics for all respiratory infections."Moreover, "CF patients' lung function, as measured by forcedexpiratory volume, improved 5.8 percent when PulmozymeDNase was used once a day ... and was maintained throughoutthe period of the study."

Fewer respiratory tract infections should lead to a reducedfrequency and duration of hospital stays, according to DavidStone, an analyst with Cowen & Co. Those hospital stays areexpensive, he said. A young CF patient in the first five to 10years of life might ring up $5,000 to $10,000 in treatmentcharges annually -- most going to antibiotic therapy andhospitalization. By the time they're teenagers, Stone said, theannual costs are up to $15,000-$20,000, and in the last five to10 years of life, the costs of care are up to $45,000 per year.(Median survival for CF patients is 29 years. It affects about30,000 Americans, almost 3,000 Canadians and 20,000Europeans annually.)

Stone predicted that Genentech will gain marketing approval inthe U.S. for Pulmozyme by the fourth quarter of this year. Heexpects "very rapid market penetration for the product and fullmarket penetration two years after product launch, at whichtime it may account for as much as half of Genentech's totalproduct sales."

Denise Gilbert, an analyst with Smith Barney, is alsoenthusiastic about Pulmozyme's prospects. She foreseesproduct approval in mid-to-late 1994, and a maximum U.S.market potential for treating CF of $250 million.

Gilbert predicted that in 1995 sales of Pulmozyme will accountfor as much as 28 percent of the company's total product sales(the other products being growth hormone and tissueplasminogen activator). And by 1998, when Pulmozyme shouldbe approved for treating chronic bronchitis, as well as cysticfibrosis, Gilbert sees Pulmozyme capturing 33 percent ofGenentech's sales.

"This is the first of a fairly large product pipeline that comesbehind it," Gilbert told BioWorld. "Pulmozyme is the productthat restarts Genentech's earnings growth."

-- Jennifer Van Brunt Senior Editor

(c) 1997 American Health Consultants. All rights reserved.

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