GlaxoSmithKline (GSK) announced that the United States Food and Drug Administration (FDA) granted accelerated approval for Promacta (eltrombopag) for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. The approval is a significant milestone for eltrombopag and the ITP community, as eltrombopag is the first oral thrombopoietin (TPO) receptor agonist approved for adult patients with chronic ITP. GSK expects eltrompopag to be available next week.
"Preventing platelet destruction has always been the primary means of treating patients with ITP. Recent advances, like the clinical trials of eltrombopag, show that increasing the production of platelets may also play a significant role in treating this disorder," said James Bussel, director of the Platelet Disorders Center, Children's Cancer and Blood Foundation Division of New York Presbyterian/Weill Cornell Medical Center. "The entire ITP community, including physicians, patients and their families may benefit from this shift in thinking and these innovative new treatments."
The new drug application for eltrombopag was supported by the largest database of randomized clinical trial information on investigational therapies for chronic ITP patients. The indication is based on data from two pivotal studies in the short-term treatment and one ongoing long-term treatment study of patients with chronic ITP.
"The approval of eltrombopag affirms our commitment to haematology and oncology research and demonstrates our dedication to developing truly innovative therapies for patients," said Paolo Paoletti, senior vice president of the Oncology Medicine Development Center at GSK. "Eltrombopag is a significant step forward in helping patients and physicians better manage the challenges of chronic ITP."
The approval of eltrombopag was supported by a unanimous decision by the FDA's Oncology Drugs Advisory Committee (ODAC) on May 30, 2008, in which the panel voted, 16-0 that eltrombopag demonstrated a favourable risk-benefit profile for the short-term treatment of patients with chronic ITP.
GSK is launching Promacta Cares, a single source of information, education and support for healthcare professionals and patients. Prescribers and pharmacies must enrol in Promacta Cares before they can prescribe or dispense eltrombopag. Similarly, patients are required to enrol in Promacta Cares before they can receive the drug. This programme was created in accordance with the FDA's requirements to help assure the appropriate and safe use of eltrombopag, while minimizing risks, including the risk of hepatoxicity. Promacta Cares is part of an ongoing collaboration between GSK and the FDA to provide a format for appropriate additional data collection.
Additionally, Promacta Cares has an optional component; reimbursement support for the uninsured and under-insured as part of GSK's ongoing commitment to providing needed medicines to those who may benefit from assistance. Knowledgeable consultants are available to assist patients and healthcare professionals with disease and product-related questions and to help in obtaining the product.
Chronic ITP is a disorder marked by increased platelet destruction and/or inadequate platelet production in the blood, which causes an increased risk of bruising and bleeding.
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.