Pulmozyme, Genentech Inc.'s recombinant DNase for treatingcystic fibrosis, has been approved for marketing in both theU.S. and Canada. The U.S. FDA and the Canadian HealthMedicines Branch granted marketing licenses for the producton Thursday.
Pulmozyme has already been approved for marketing inAustria, Sweden and New Zealand, and on Dec. 17 the EuropeanCommittee for Proprietary Medicinal Products (CPMP)unanimously recommended approval of Pulmozyme (the CPMPmember states still must grant individual marketingauthorizations).
Pulmozyme is the first new therapy for treating CF, a fatalgenetic disease, to come along in decades. It acts by liquefyingthe thick mucus that is produced in the lungs by patients withCF, which affects about 30,000 Americans, 3,000 Canadians andmore than 20,000 Europeans.
In clinical trials the drug has been shown to reduce the rate ofrespiratory infection in CF patients by 27 percent, as well assignificantly improve their lung function. The trials alsodemonstrated that use of Pulmozyme reduces hospitalizationsand other related medical costs; importantly, it also improvesthe quality of life in patients with mild to moderate pulmonarydysfunction.
The FDA approved the drug just nine months after Genentech(NYSE:GNE) filed its product license application (PLA). FDA'sPulmonary Allergy Drugs Advisory Committee unanimouslyrecommended approval of the drug on Aug. 9.
Genentech of South San Francisco, Calif., filed for productapproval in Canada and Europe in late March, as well.
The FDA approved Pulmozyme (dornase alfa) for use in cysticfibrosis (CF) patients in stable condition over 5 years of agewhose lung function is greater than 40 percent (as measuredby forced vital capacity). The recommended dose of the drug is2.5 milligrams once daily for the defined patient population(although the FDA advisory committee did suggest that twice-daily schedules for patients outside the defined parameters beincluded in the clinical pharmacology section of the packageinsert).
The product should be on the market by mid-January.Genentech has already manufactured enough drug to meet thedemand worldwide, according to Jim Weiss, manager ofcorporate communications. "There will be no shortage," Weissemphasized, especially since the product has a shelf life of 12-14 months.
Although Genentech has not yet set the final price of theproduct, several analysts have zeroed in on a cost of $10,000for a year's regimen (once daily in 2.5 milligram doses). Thistallies to $93 million in revenues in 1994, according to RobFein, an analyst with Nomura Securities International. AndDavid Molowa, an analyst with Bear Stearns Co., is only slightlymore conservative, predicting that product sales worldwidewill reach $90 million in 1994 (with 20 percent marketpenetration) and $170 million in 1995 (with 35 percent marketpenetration).
Molowa said that sales of Pulmozyme might run even with orslightly better than Genentech's growth hormones (Protropinand Nutropin combined), but still fall short of sales of its clot-buster Activase in 1994.
But both analysts see significantly greater sales long-termbased on off-label use in non-CF patients, especially those withchronic bronchitis and other chronic pulmonary diseases. Infact, Molowa predicted that Pulmozyme will ultimately beGenentech's single biggest product, with sales in excess of $500million.
Genentech's stock gained $1.13 a share on Thursday, closing at$49.50.
-- Jennifer Van Brunt Senior Editor
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