PromactaTreatment for Idiopathic (Immune) Thrombocytopenic Purpura
Update: Promacta Now FDA Approved - November 20, 2008
Promacta Recommended by FDA ODAC
Promacta (eltrombopag) Receives Unanimous Recommendation by FDA Advisory Panel
CHICAGO, May 30, 2008 /PRNewswire-USNewswire/ -- GlaxoSmithKline today announced that the United States Food and Drug Administration's Oncology Drugs Advisory Committee (ODAC) unanimously voted, 16-0, that Promacta (eltrombopag) demonstrated a favorable risk-benefit profile for the short-term treatment of patients with chronic idiopathic thrombocytopenic purpura (ITP). The FDA advisory committee was held onsite at the 2008 American Society of Clinical Oncology (ASCO) Annual Meeting.
The advisory committee reviewed studies evaluating the safety and efficacy of eltrombopag in the short-term setting. Clinical data were presented and discussed that support eltrombopag increased platelet counts and reduced bleeding.
"Chronic ITP is a condition, which can have serious complications and be life threatening. Two of the most fundamental endpoints are crucial to managing this disease -- improved platelet counts and decreased bleeding. If approved, this would make the drug a potential new clinical option that can address a true unmet medical need," said Paolo Paoletti, M.D., Senior Vice President of the Oncology Medicine Development Center at GSK. "An FDA approval for eltrombopag would represent an important development for these patients and would make the medication the first oral treatment of its kind for this disease. We look forward to working with the agency to achieve that goal."
In addition to today's recommendation, the FDA recently granted eltrombopag orphan drug designation for this indication. Orphan designation 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.(1)
Eltrombopag is an investigational, once-daily oral treatment developed to induce the production of cells in the bone marrow to increase platelets, which are critical in minimizing the incidence of bleeding in chronic ITP.
Eltrombopag, a novel oral thrombopoeitin (TPO) receptor agonist, if approved, would be the first oral short-term treatment of previously treated patients with chronic ITP to increase platelet counts and reduce or prevent bleeding.
In the pivotal studies, the most common adverse events observed in patients taking eltrombopag were headache, nasopharyngitis, and nausea.
The NDA submission is supported by the largest database of clinical trial information on investigational therapies for chronic ITP patients. Two pivotal trials (one Phase III trial and one Phase II trial), were submitted to support the filing.
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.(2,3) There are estimated to be approximately 60,000 individuals diagnosed with chronic ITP in the U.S.(4) People with chronic ITP often bleed from small blood vessels causing bruises, nosebleeds or even fatal gastrointestinal or intra cerebral bleeds, although these are rare.(3)
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. Eltrombopag was discovered as a result of research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals . It is being developed by GlaxoSmithKline. Eltrombopag is an investigational compound that has not received regulatory approval in any market for any indication at this time.
GlaxoSmithKline -- one of the world's leading research-based pharmaceutical and healthcare companies -- is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For company information, visit GlaxoSmithKline at www.gsk.com.
Under the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995, GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Factors that may affect GSK's operations are described under 'Risk Factors' in the 'Business Review' in the company's Annual Report on Form 20-F for 2007.
Promacta is a registered trademark of GlaxoSmithKline group of companies and is the proposed trade name in the United States.
To access the latest GSK news, visit http://us.gsk.com/
US Media inquiries: Jeff McLaughlin 1 919 483 2839 Mary Anne Rhyne 1 919 483 2839 Nancy Pekarek 1 215 751 7709 US Analyst/Investor inquiries: Frank Murdolo 1 215 751 7002 Tom Curry 1 215 751 5419
(1) US Food and Drug Administration. The Orphan Drug act (as amended). Accessed May 2008. http://www.fda.gov/orphan/oda.htm
(2) National Heart, Lung, and Blood Institute. Diseases and Conditions Index. http://www.nhlbi.nih.gov/health/dci/Diseases/Itp/ITP_WhatIs.html. Accessed November 12, 2007.
(3) Cines DB, Blanchette V. Idiopathic thrombocytopenic purpura. N Engl J Med. 2002;364: 995-1008.
(4) Feudjo-Tepie M, Robinson N, Bennett D. Prevalence estimates of adult chronic idiopathic thrombocytopenic purpura (ITP). 2008 doi: 10.1111/j.1538-7836.2008.02911.x.
CONTACT: Jeff McLaughlin or Mary Ann Rhyne, +1-919-483-2839, or NancyPekarek, +1-215-751-7709
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Posted: May 2008
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