RecAAT has already received orphan drug designation in the U.S. for cystic fibrosis indication in March 1998, and for congenital emphysema indication in June 1999. The results demonstrated that recAAT was efficiently delivered into the deep lung, and that by using a higher concentration the nebulization time was significantly shortened. Pivotal Phase III trials for recAAT in the aerosol formulation are expected to begin in the third quarter of 2001. Alpha-1 Antitrypsin Deficiency AAT deficiency is a potentially lethal hereditary disease, which can lead to a form of emphysema, affecting more than 200,000 people worldwide. Following the development of emphysema, this disease is characterized by shortness of breath, wheezing, coughing, and recurrent lung infections.
Many symptomatic patients die from pulmonary causes in their fifth and sixth decades of life.
Under the agreement, Bayer is responsible for, and bears the costs of, clinical development and marketing. PPL is responsible for exclusive product manufacturing. Best known for its flagship product, Bayer Aspirin, Bayer Corporation produces a broad range of health care, life sciences, and chemical products that help diagnose and treat diseases, purify water, preserve local landmarks, protect crops, advance automobile safety and durability, and improve people's lives. The company employs 23,200 people. PPL Therapeutics is a biopharmaceutical company which is one of the world's leaders in the application of transgenic animal technologies to the development and production of human proteins for therapeutic and nutritional applications.