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First Oral Thrombopoietin Receptor Agonist Approved for Adult Patients

PHILADELPHIA, Nov. 20 /PRNewswire-FirstCall/ -- GlaxoSmithKline (NYSE:GSK) today announced that the United States Food and Drug Administration (FDA) granted accelerated approval for PROMACTA(R) (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 PROMACTA and the ITP community, as PROMACTA is the first oral thrombopoietin (TPO) receptor agonist approved for adult patients with chronic ITP. GSK expects PROMACTA to be available by the next week.

"It is very important for chronic ITP patients to have new treatment options to manage the symptoms of this serious and, at times, life-threatening disease. In addition, many people with chronic ITP are concerned about everyday activities for fear of an accidental bump or unanticipated bruise that may lead to bleeding," said Craig Conway, executive director of the Platelet Disorder Support Association. "The approval of this new product represents a promising new treatment option that offers hope to the ITP community."

Shift in Approach
"Preventing platelet destruction has always been the primary means of treating patients with ITP. Recent advances, like the clinical trials of PROMACTA, show that increasing the production of platelets may also play a significant role in treating this disorder," said James Bussel, M.D., 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 PROMACTA 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 PROMACTA affirms our commitment to hematology and oncology research, and demonstrates our dedication to developing truly innovative therapies for patients," said Paolo Paoletti, M.D., Senior Vice President of the Oncology Medicine Development Center at GSK. "PROMACTA is a significant step forward in helping patients and physicians better manage the challenges of chronic ITP."

The approval of PROMACTA was supported by an unanimous decision by the FDA's Oncology Drugs Advisory Committee (ODAC) on May 30, 2008, in which the panel voted, 16-0, that PROMACTA demonstrated a favorable 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 enroll in PROMACTA CARES before they can prescribe or dispense PROMACTA. Similarly, patients are required to enroll in PROMACTA CARES before they can receive the drug. This program was created in accordance with the FDA's requirements to help assure the appropriate and safe use of PROMACTA, while minimizing risks. 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. More information regarding PROMACTA CARES can be found by calling 1-877-9-PROMACTA or by visiting Program hours are 8:30 AM to 8:00 PM ET, Mondays through Fridays.

GSK plans to submit a Marketing Authorization Application (MAA) for eltrombopag in the treatment of chronic ITP in Europe in 2008; the proposed trade name is REVOLADE.

PROMACTA milestones
Following the New Drug Application (NDA) filing for PROMACTA in December 2007, the FDA granted the drug Priority Review in March 2008. In addition, the agency granted PROMACTA orphan drug designation for this indication. This is a special status the FDA grants to products that treat a rare disease or condition, affecting less than 200,000 individuals in the United States.

About ITP
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. There are estimated to be approximately 60,000 individuals diagnosed with chronic ITP in the U.S. People with chronic ITP often bleed from small blood vessels causing bruises, nosebleeds or even fatal intracerebral bleeds, although these are rare.

PROMACTA 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. PROMACTA was discovered as a result of a research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals (NASDAQ:LGND). It was developed by GlaxoSmithKline.

The compound is also being studied for thrombocytopenia associated with chronic hepatitis C virus, chronic liver disease and oncology-related thrombocytopenia.

Source: GlaxoSmithKline

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