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December 21st, 2005 FDA grants orphan-drug status to Uniplas The US Food and Drug Administration (FDA) announced December 12th that it has granted Octapharma USA Inc. orphan drug status for Uniplas (solvent/detergent [S/D] treated non-blood group specific human coagulation active plasma) for the treatment of thrombotic thrombocytopenic purpura (TTP). The orphan drug designation will enable Octapharma to commence a clinical trial program for Uniplas in TTP patients with a view to obtaining TTP is a rare blood condition characterized by the formation of small clots (thrombi) within the circulation, which results in the consumption of platelets and thus a low platelet count (thrombocytopenia) A recent study estimated the incidence of TTP as 11.29 cases per million population, meaning that there will be approximately 3,300 cases each year in the United States.1 TTP is thought to be caused by a deficiency of von Willebrand factor (VWF) cleaving protease - a metalloprotease enzyme (ADAMTS 13). Individuals with TTP synthesise VWF normally, but its breakdown is defective. The symptoms of TTP may at first be subtle- malaise, fever, headache & diarrhea. As the condition progresses clots form within blood vessels & platelets are consumed. Bruising and bleeding results. Bruising often takes the form of purpura. The most common site of bleeding are the nose or gums, however, larger bruises (ecchymoses) may also develop. Clots formed within the circulation can temporarily disrupt local blood supply. TTP preferentially affects the blood vessels of the brain and kidneys. Thus a patient may experience headache, confusion, difficulty speaking, transient paralysis, numbness or even fits. If not treated correctly, the mortality rate is over 90%.2 Plasma exchange is the most important component of treatment for TTP. It involves the removal of a patient's plasma and its replacement by donor plasma to remove circulating antibody against VWF-cleaving protease and provide plasma with normal VWF-cleaving protease activity. The procedure usually needs to be performed daily for at least five days. Sometimes many more procedures are required. Uniplas will undergo studies to evaluate its potential as an alternative to the current treatment of TTP which involves the administration of non-virus inactivated, single donor, blood-group specific plasma or cryo-poor plasma (cryosupernatant). Uniplas is a unique, product developed and patented by Octapharma. It is produced from a optimized mixture of blood group A, B and AB donor plasma resulting in a product with very low levels of anti-A, anti-B antibodies that can potentially be given to recipients, regardless of their ABO blood group. Additionally, the product also undergoes pathogen inactivation by the solvent/detergent method to reduce the risk of transfusion transmitted disease. The pooling of plasma to produce Uniplas also results in dilution of specific antibodies (HLA) which virtually eliminates the risk of transfusion-associated lung injury (TRALI). Orphan-drug designation was designed to promote the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions, specifically those that affect less than 200,000 people in the References 1. Terrell DR, Williams LA, Vesely SK, Lämmle B, Hovinga JAK & George JN. The incidence of thrombotic thrombocytopenic purpura-hemolytic uremic syndrome: all patients, idiopathic patients, and patients with severe ADAMTS-13 deficiency. J Thromb Haemost. 2005, 3;7:1432-1436 2. 3. OOPD Frequently Asked Questions. http://www.fda.gov/orphan/faq/index.htm Please note that clicking on these links will take you outside of the Octapharma web site) For further information on Uniplas please contact: Marc Maltas International Business Manager - Intensive Care Seidensreasse, 2 8853 Lachen Switzerland Tel (+ 41) 55 451 21 24 © Octapharma AG, 2005
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