European Medicines Agency Supports Maintaining Orphan Designation for IDELVION®
MARBURG, Germany — 05 April 2016
CSL Behring announced today that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) supports maintaining the orphan designation for IDELVION® [Coagulation Factor IX (Recombinant), Albumin Fusion Protein]. IDELVION® is CSL Behring’s novel, long-acting recombinant albumin fusion protein for treating hemophilia B. Recently, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended granting marketing authorization for IDELVION® in the European Union, for the treatment and prophylaxis of bleeding in patients with Haemophilia B (congenital factor IX deficiency).
To qualify for orphan designation, a medicinal product is intended for the safe and effective treatment, prevention or diagnosis of life-threatening or chronically debilitating diseases that affect not more than 5 in 10,000 people throughout Europe. Sponsors who obtain orphan designation benefit from market exclusivity once the medicine is on the market.
CSL Behring engineered IDELVION® (also known as rIX-FP) to extend the half-life of recombinant factor IX through fusion with recombinant albumin. CSL Behring selected recombinant albumin as its fusion partner due to its long physiological half-life. Additionally, albumin has been shown to have a good tolerability profile, low potential for immunogenic reactions and a well-known mechanism of clearance. The cleavable linker connecting recombinant factor IX and recombinant albumin has been specifically designed to preserve the native function of the coagulation factor in the fusion protein, while benefiting from recombinant albumin’s long physiological half-life.
IDELVION® is approved in the United States and Canada. Regulatory agencies in Australia, Switzerland and Japan are also currently reviewing CSL Behring’s marketing applications for IDELVION®.
About CSL Behring
CSL Behring is a global biotherapeutics leader which is driven by its promise to save lives. Focused on serving patients’ needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.
CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL), headquartered in Melbourne, Australia, employs more than 16,000 people with operations in more than 30 countries. For more information visit www.cslbehring.com and follow us on www.Twitter.com/CSLBehring. For more information about CSL Behring visit www.CSLBehring.com or follow us at www.Twitter.com/CSLBehring.