Talecris Biotherapeutics : Orphan Drug Designation for Aerosolized Form of Alpha-1 Antitrypsin (AAT) From the European Commission

July 24, 2008 — Talecris Biotherapeutics, Inc. announced that the European Commission followed an opinion of the Committee for Orphan Medicinal Products (COMP) and has granted orphan drug designation to Talecris’ alpha₁ proteinase inhibitor [human] for inhalation use in the treatment of congenital Alpha₁-Antitrypsin Deficiency (also known as AAT Deficiency or Alpha-1). Currently, there are no approved aerosolized treatments available for augmentation therapy for AAT Deficiency...
...About Alpha₁-Antitrypsin Deficiency
Severe Alpha₁-Antitrypsin Deficiency, also known as AAT Deficiency or Alpha-1, is an inherited disorder that causes a significant reduction of the serum concentration of the naturally occurring protein AAT. It is most common in the Caucasian population of northern Europe and North America. AAT Deficiency is also the most common cause of genetic liver disease in children. In adults, AAT Deficiency is associated with a predisposition for severe chronic obstructive pulmonary disease (COPD), especially early-onset emphysema causing disability and premature death. An estimated 100,000 people in the United States have AAT Deficiency. In Europe, the prevalence of AAT Deficiency is about 2.5 in 10,000 individuals... Talecris Biotherapeutics' Press Release -