GENENTECH’s PULMOZYME COULD BE REVIEWED AT AUGUST MEETING

Executive Summary

GENENTECH's PULMOZYME COULD BE REVIEWED AT AUGUST MEETING of FDA's Pulmonary-Allergy Drugs Advisory Committee. Although the agenda for the committee's next meeting has not been made available yet, Genentech's recombinant DNase product is a possible topic based on FDA's effort to move that product through the review process rapidly. The committee is scheduled to meet Aug. 9- 10. DNase is being pursued as a treatment for pulmonary symptoms of cystic fibrosis. Genentech said it has discussed with FDA the possibility of bringing Pulmozyme before the committee in August, but the company has not received written confirmation that it will be on the agenda. The committee review would come five months after the filing of the Pulmozyme PLA, which is consistent with other rapid reviews recently for important new therapies such as Bristol-Myers Squibb's Taxol. From filing to committee review of Taxol took four months. Genentech filed the PLA for Pulmozyme in March ("The Pink Sheet" April 5, In Brief). The application is being reviewed jointly by the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research. The PLA for Pulmozyme contains Phase III data on 968 cystic fibrosis patients treated for six months with the drug. The trials measured improvements in pulmonary function, well being and quality of life as well as reduction in concurrent therapy with antibiotics and number of days of hospitalization ("The Pink Sheet" Feb. 8, In Brief). At a June 21 meeting on computer-assisted applications sponsored by the Institute on International Research, Genentech's Kevin Dwyer described FDA's proactive interest in expediting the review of Pulmozyme. Dwyer said that at a routine PLA format meeting in September 1992, the agency "surprised" Genentech representatives by requesting electronic submissions of certain parts of the application to assure a rapid review of the drug. Genentech had not filed a computer-assisted PLA (CAPLA) initially because "we were not set up to do an electronic submission," Dwyer explained. In addition, the company had to gear its submission to different markets. "Due to our interactions with [majority shareholder] Roche Holdings at the time we were required to file simultaneously in Europe, as well as Canada," Dwyer said. The company eventually filed a partial CAPLA with FDA, including electronic text files of Phase II/III data and integrated summaries of safety and efficacy. Genentech had used a Xerox software system in preparing the application, Dwyer said, but the company was required to convert all the information to Microsoft, the system used by FDA. Dwyer suggested that companies anticipating expedited review for a product should be prepared to file a full CAPLA.