FDA grants Talecris Biotherapeutics orphan drug designation for Alpha1-Proteinase Inhibitor to treat AAT deficiency

Feb 5 2010

Talecris Biotherapeutics, Inc. (Nasdaq: TLCR) announced today that it was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the development of an aerosol formulation of Alpha₁-Proteinase Inhibitor (Human, A1PI) to treat congenital alpha₁-antitrypsin (AAT) deficiency. AAT deficiency is a chronic, hereditary condition that increases the risk of certain diseases, especially emphysema, which typically emerges in the fourth decade of life. Currently, there are no approved, inhaled treatments available for the treatment of AAT deficiency.

“This orphan drug designation will allow us to move forward with developing an alternative method of delivering augmentation therapy for patients who prefer an inhaled mode of administration.”

Orphan drug designation is granted to companies to encourage the development of treatments that prevent, diagnose or treat rare, life-threatening or chronic illnesses that affect fewer than 200,000 people per year in the U.S. The designation provides incentives such as tax credits and potentially seven years of market exclusivity to companies willing to support the costly research and development programs associated with developing specialized drugs for a small population of individuals.

The initiative helps to give patients suffering from rare diseases access to the same quality of treatment as other patients. Talecris received a similar orphan drug designation for the aerosolized form of AAT from the European Commission in June of 2008.

Talecris is the manufacturer of PROLASTIN^® (Alpha₁-Proteinase Inhibitor [Human]) an intravenous therapy that is indicated for chronic augmentation therapy among individuals who have AAT deficiency. Individuals with AAT deficiency have low serum concentrations of the A1PI protein in their blood and lungs. Augmentation therapy is administered to raise levels of the A1PI protein.

“Talecris is committed to helping patients with rare diseases for whom few treatment options exist,” said Lawrence D. Stern, chairman and CEO of Talecris. “This orphan drug designation will allow us to move forward with developing an alternative method of delivering augmentation therapy for patients who prefer an inhaled mode of administration.”

An important part of Talecris’ aerosol development program is the exclusive partnership between Talecris and Activaero Technologies (www.activaero.de/en.php), an industry leader in controlled breathing technologies for inhaled therapeutic agents. Activaero’s AKITA^2® APIXNEB inhalation system has demonstrated consistently high drug deposition to the central and peripheral regions of the lungs in patients with AAT deficiency, regardless of disease severity.