Talecris Biotherapeutics, Inc. has announced 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(R) 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.
