Aventis Behring Submits Application for FDA Approval of New Intravenous Alpha-1 Antitrypsin to Treat Hereditary Emphysema 

Monday January 6, 2003 8:00 am ET 

KING OF PRUSSIA, Pa.--(BUSINESS WIRE)--Jan. 6, 2003--Aventis Behring L.L.C. announced today that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of an intravenous form of Alpha1- Proteinase Inhibitor IV [Human] (A1-PI (H) IV), for use as chronic augmentation and maintenance therapy in individuals with Alpha1-Antitrypsin Deficiency (AAT Deficiency) or hereditary emphysema, a life-threatening genetic disorder. 

The submission of the BLA for the treatment of AAT Deficiency is based on the outcome of clinical trials. The primary endpoints of the phase III 44-subject trial were successfully met and the study data suggest that the treatment is effective in maintaining serum levels of A1-PI above the threshold, over a 
24-week period. These levels are considered by the Alpha-1 medical community to be protective against lung damage. In addition, the trial results indicate that A1-PI is delivered to the lower lung and is able to bind to neutrophil elastase, which is thought to cause the lung damage associated with AAT Deficiency. The trial also suggests that the product is comparable to existing marketed AAT replacement therapy. 

This product previously received Fast Track designation from the FDA in December 2000. Under the FDA Modernization Act of 1997, the FDA facilitates and expedites the development and review of the application for the approval of a new drug if it is intended for the treatment of a serious or life-threatening condition and has the potential to address an unmet medical need. 

"We are pleased with the submission of our BLA and are excited to be one step closer to regulatory approval of our intravenous Alpha1-Antitrypsin therapy. The Alpha-1 Community has been anxiously awaiting the entry of new products into this field, and we are gratified that we are in a position to try to help meet the demands of the community," said Dr. Ruedi Wager, Chief Executive Officer, Aventis Behring. 

"A critical short supply of the therapy used to treat the lung disease associated with Alpha-1 has prevented many patients who are eligible from having access to care. We commend Aventis Behring for its efforts in developing a second product for this underserved population and we support their commitment to expedite this much needed product to ensure adequate supply for patients everywhere." said John W. Walsh, President & CEO, Alpha-1 Foundation.