GlaxoSmithKline (GSK) presented positive safety and efficacy results from RAISE (RAndomised placebo-controlled ITP Study with Eltrombopag), a phase-III study of Promacta/Revolade (eltrombopag) in adults with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who had received one or more prior ITP therapies. Patients receiving eltrombopag were eight times more likely than those on placebo to maintain platelet counts between 50-400,000/mL during a six-month treatment period, thereby reducing patients' bleeding symptoms and their need for concomitant and rescue ITP treatments. These data were presented at the 50th Annual Meeting of the American Society of Haematology (ASH), December 6-9, 2008, in San Francisco, CA.
"Eltrombopag is the first approved agent to show that generating platelets can be achieved and maintained with an oral therapy," said Paolo Paoletti, senior vice president of Oncology R&D, GSK. "With the continued emergence of GSK in oncology, we want patients and physicians to continuously benefit from our dedication to developing truly innovative treatments that can help improve patients' lives. Eltrombopag is a great example of this commitment."
Eltrombopag received accelerated approval by the FDA on November 20 as a thrombopoietin receptor treatment for patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Eltrombopag should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. Eltrombopag should not be used in an attempt to normalise platelet counts. GSK submitted a Marketing Authorisation Application (MAA) for eltrombopag in Europe and other international markets in early December 2008.
Chronic ITP is a disorder marked by increased platelet destruction and/or inadequate platelet production in the blood, which causes an increased risk of bleeding.1 Approximately 60,000 individuals in the US have the disorder.
"Patients with chronic ITP often have a difficult time managing their disease. They may experience excessive bruising, bleeding and sometimes more serious haemorrhages that can rarely be fatal. Until recently, ITP patients have had few options well demonstrated to be effective in the long term," said James Bussel, director of the Platelet Disorders Center, Children's Cancer and Blood Foundation Division of New York Presbyterian/Weill Cornell Medical Center. "As the RAISE study demonstrates, eltrombopag is an important new oral treatment option for ITP patients that is effective in maintaining a haemostatic platelet response."
Eltrombopag is an oral, non-peptide thrombopoietin receptor agonist that has been shown in pre-clinical research and clinical trials to stimulate the proliferation and differentiation of megakaryocytes, the bone marrow cells that give rise to blood platelets.