Talecris Biotherapeutics’ aerosol formulation of Alpha1-Proteinase Inhibitor (Human, A1PI) to treat congenital alpha1-antitrypsin (AAT) deficiency, was granted orphan drug designation by the FDA. AAT deficiency is a chronic, hereditary condition that increases the risk of certain diseases, particularly emphysema. Currently, there are no approved, inhaled treatments available to treat AAT deficiency.
Orphan drug designation is granted to encourage the development of treatments that prevent, diagnose or treat rare, life-threatening or chronic illnesses and provides incentives such as tax credits and potentially seven years of market exclusivity.
Talecris manufactures Prolastin (Alpha1-Proteinase Inhibitor [Human]), an intravenous therapy that is indicated for chronic augmentation therapy in patients with AAT deficiency. Individuals with AAT deficiency have low 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 chief executive officer 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.”
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