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 -

Talecris Biotherapeutics, 20th Anniversary of Launch of Prolastin

(Feb. 21, 2008) — Talecris Biotherapeutics, Inc., a leading global biotherapeutics and biotechnology company, is celebrating the 20th anniversary of the launch of Prolastin^® (Alpha1-Proteinase Inhibitor [Human]). Prolastin is the market leader among alpha1-antitrypsin (AAT) augmentation therapies and is used to treat people with AAT deficiency, or Alpha-1, who have developed emphysema as a result of low circulating levels of AAT. AAT deficiency is an inherited condition thought to affect as many as 150,000 people in North America and Europe, although only an estimated five percent have been diagnosed. Prolastin was the first plasma-derived AAT augmentation therapy to receive approval from the U.S. Food and Drug Administration (FDA). Approved in December 1987, and launched in February 1988, Prolastin was the first FDA-approved product for AAT augmentation therapy for Alpha-1 patients and remained so for nearly 17 years...
...Before Prolastin was approved, people with Alpha-1 received only supportive therapy for their pulmonary symptoms but did not have access to a specific therapy that raises AAT (anti-elastase) levels above the protective threshold to preserve lung function... Talecris' Press Release -