GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Talecris Receives EU Orphan-Drug Designation for Aerosolized AAT

[Aerosolized AAT should not be confused with Prolastin, an injected form of AAT marketed by Talecris that is the main product GTC hopes to take share from (#msg-27011864). This PR says that Talecris will seek FDA orphan status for aerosolized AAT; Kamada already has an orphan designation in both the EU and US (#msg-27079832). Please see #msg-25174719 and #msg-25848126 for musings on how GTC may eventually be able to break the orphan designation for inhaled AAT from Talecris or Kamada by virtue of having a recombinant product.]

http://biz.yahoo.com/bw/080724/20080724005411.html

›Thursday July 24, 9:15 am ET

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Talecris Biotherapeutics, Inc. announced today that the European Commission followed an opinion of the Committee for Orphan Medicinal Products (COMP) and has granted orphan drug designation to Talecris’ alpha-1 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.

By providing incentives to the pharmaceutical industry, the EU legislative framework for orphan medicines encourages the development of products intended for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions impacting no more than 5 in 10,000 people in the European Union. The initiative helps to give patients suffering from rare diseases access to the same quality of treatment as other patients.

Through this designation, the European Medicines Agency (EMEA) will provide to Talecris ten years of market exclusivity if the product is the first to be approved for marketing in the European Union. In addition, under this designation, EMEA will provide Talecris with clinical development assistance and reduced regulatory fees. The Food and Drug Administration (FDA) has a similar designation for products developed to treat orphan diseases in the United States. Talecris intends to seek orphan drug designation in the US.

Larry Stern, chief executive officer of Talecris Biotherapeutics, said, “The European Commission’s designation provides another promising development milestone for Talecris in the expansion of our alpha-1 franchise—a pursuit that reflects our ongoing commitment to offer clinical advancements that will benefit patients.”

An important part of this alpha-1 antitrypsin aerosol development program is the exclusive partnership between Talecris and Activaero Technologies (http://www.activaero.de/en.php), an industry leader in controlled breathing technologies for inhaled therapeutic agents, for its AKITA2® APIXNEB inhalation system. This system has demonstrated consistently high drug deposition to the central and peripheral regions of the lungs in patients with AAT Deficiency independent of disease severity. For more information on Talecris Research and Development programs, please visit http://www.talecris.com.

Talecris Biotherapeutics is a global biotherapeutic and biotechnology company that discovers, develops and produces critical care treatments for people with life-threatening disorders in a variety of therapeutic areas including immunology, pulmonology, and hemostasis. Talecris is proudly building upon a 60-year legacy of innovation and a commitment to improving the lives of people who rely on its therapeutic products. With an emphasis on scientific inquiry and technological excellence, Talecris is expanding its current portfolio of products, programs, and services through its own world-class product development organization as well as through strategic initiatives that leverage its strengths with those of its partners.

Talecris, with revenues of approximately $1.2 billion in 2007, is headquartered in biotech hub Research Triangle Park, N.C., and employs more than 4,000 talented people worldwide.

About Alpha1-Antitrypsin Deficiency

Severe Alpha-1-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.‹